US Market News
3週前
Jazz Pharmaceuticals Delivers Extensive Late-Breaking Data at SLEEP 2026, Demonstrating Real-World Impact of Xywav® (calcium, magnesium, potassium, and sodium oxybates) on Patient Outcomes Across Narcolepsy and Idiopathic HypersomniaJune 16, 2026 4:05 PM
PR Newswire (US) New late-breaking data presentations underscore Jazz's significant progress in advancing holistic understanding of narcolepsy and idiopathic hypersomnia, including novel real-world evidence of meaningful changes in cardiometabolic risk markers following Xywav initiationFor U.S. media and investors onlyDUBLIN, June 16, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced 11 late-breaking presentations at SLEEP 2026, including notable new research showcasing the comprehensive treatment outcomes of Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution in patients with narcolepsy and idiopathic hypersomnia (IH). These late-breaking presentations advance scientific understanding of individualized dosing regimens and the broader cardiometabolic impacts of reduced sodium exposure in patients living with these debilitating conditions."Treating rare sleep conditions like narcolepsy and idiopathic hypersomnia requires a holistic approach that extends beyond immediate symptoms. Because these patients face greater cardiovascular risk, managing sodium intake is a critical component of their care," said Logan Schneider, M.D., adjunct clinical associate professor of sleep medicine, Stanford Sleep Center and Consultant Neurologist, Stanford/VA Alzheimer's Center. "These new data from Jazz continue to build on our understanding of the relationship between sleep architecture and patient-reported sleep and improvements in patient-reported daytime symptoms, as well as how sodium reduction directly impacts patient well-being and considerations when personalizing therapy.""The breadth and rigor of the evidence we are presenting at SLEEP 2026 reflects years of dedicated scientific inquiry into narcolepsy and idiopathic hypersomnia," said Jessa Alexander, Ph.D., neuroscience therapeutic area head, global medical and scientific affairs of Jazz Pharmaceuticals. "Jazz is advancing the scientific understanding of these rare sleep conditions with novel data which demonstrates the potential of Xywav's individualized dosing optimization and the impact of its low-sodium formulation within the context of elevated cardiovascular and cardiometabolic risk. Our research is illuminating the transformative potential of Xywav to deliver outcomes which shape treatment paradigms and have the potential to improve the quality of life for patients living with these conditions."Key findings from these late-breaking presentations provide insights into the complex landscape of sleep disorders, across key areas such as:Xywav Individualized Dosing Supports Personalized Treatment Regimens (P-41, Posters #528 and #529)
New analyses demonstrate that the individualized dosing regimens Xywav allows can be uniquely tailored for patients with narcolepsy or IH. A post-hoc analysis from the Phase 4, prospective, single-arm, open-label DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) study shows how incremental dose adjustments can yield a variety of once- or twice-nightly regimens based on efficacy and tolerability of the individual patient. Within the study cohorts, the mean (range) time to reach a stable dose was 42.1 (14-62) days for idiopathic hypersomnia and 41.8 (12-67) days for narcolepsy. Reinforcing the real-world applicability of this approach, data from the Xywav REMS program, which analyzed over 13,000 patients, demonstrated the feasibility of individualized, patient-centered care enabled by Xywav's oral solution formulation when clinically warranted.Xywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy and for the treatment of IH in adults. The Xywav label recommends a nightly dosage of 6-9 grams per night.The Impact of a Low-Sodium Formulation Option on Long-Term Cardiometabolic Health (P-41, Posters #526 and #527)
New data showcases that Xywav's low-sodium formulation extended health benefits beyond the core symptoms of narcolepsy and IH, offering meaningful health gains for patients managing the long-term consequences of these conditions. Secondary results from the open-label, single-arm Phase 4 XYLO study indicate that patients with narcolepsy who switched from high-sodium oxybate to Xywav reported qualitative improvements in symptoms associated with sodium/fluid imbalance, such as edema, diaphoresis, and nocturia, highlighting the clinical benefits of lowering sodium exposure. Moreover, a novel retrospective analysis using electronic health records showed decreases in lipid biomarkers associated with cardiovascular/cardiometabolic (CV/CM) risk, including non-high-density lipoprotein (HDL) cholesterol and triglycerides after Xywav initiation, suggesting risk mitigation for CV/CM outcomes in this patient population. Notably, among the subset of patients with baseline high-sodium oxybate use, initiation of Xywav was associated with additional CM benefits, including a decrease in office-based systolic blood pressure and an increase in HDL cholesterol.The full abstracts will be available online at: sleepmeeting.org/abstract-supplementsAbout Narcolepsy
Narcolepsy is a chronic, debilitating neurologic sleep disorder characterized by the inability to maintain continuous sleep at night and sustained wakefulness throughout the day. This leads to symptoms that can include fragmented or disrupted nighttime sleep, excessive daytime sleepiness, and cataplexy.1 Patients with narcolepsy experience severe excessive daytime sleepiness that can manifest as sleep attacks and, despite fighting the urge to sleep, may unintentionally fall asleep for short periods.2,3?These sleep attacks may happen at inappropriate or potentially dangerous times such as during driving, cycling, eating, or mid-conversation.4 There is no cure for narcolepsy; the symptoms are lifelong and have a substantial negative impact on a person's ability to function psychologically, socially and professionally.5?Patients with narcolepsy are at increased risk for?hypertension, cardiometabolic morbidity,?stroke, myocardial infarction, heart failure, cardiac arrest, and death.6,7,8,9 As narcolepsy is a chronic condition that requires lifelong treatment, early access to an effective treatment can help reduce the impact of narcolepsy symptoms on a person's physical and mental health, and long-term impacts of the treatment on cardiovascular health should be considered.5 About Idiopathic Hypersomnia?
Idiopathic hypersomnia (IH) is a debilitating, neurologic sleep disorder that goes beyond chronic excessive daytime sleepiness.10,11,12,13?Idiopathic hypersomnia is a 24-hour sleep disorder, and symptoms may include non-restorative sleep with or without long sleep time (main, nighttime, sleep episode of more than 9 hours, or a sleep duration of 11 hours or longer over a 24-hour period); severe sleep inertia (prolonged difficulty waking, with frequent reentries into sleep, confusion, and irritability); long and unrefreshing naps; cognitive impairment and brain fog, or the inability to focus for long periods of time.10,11,12,13,14?Although there are some overlapping clinical features with narcolepsy, idiopathic hypersomnia is a condition with its own specific diagnostic criteria.13,15? Idiopathic hypersomnia is an often debilitating illness that can significantly affect social, educational, and occupational functioning.16,17 In the U.S., approximately 28,000 adult patients are diagnosed with idiopathic hypersomnia annually and are actively seeking healthcare, with the diagnosed prevalence continuing to rise year over year.18 This low number of people may be due to the many difficulties in identifying and diagnosing idiopathic hypersomnia, as well as distinguishing it from other similar sleep disorders. It is estimated that far fewer patients are currently receiving pharmacological treatment for their idiopathic hypersomnia.19,20,21 About Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution
Xywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy. The Office of Orphan Product Development (OOPD) at the FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy by means of greater cardiovascular safety compared to Xyrem® (sodium oxybate) oral solution. The decision of the OOPD is based on the FDA findings that Xywav provides a greatly reduced chronic sodium burden compared to Xyrem. Xywav has 131 mg of sodium at the maximum recommended nightly dose whereas other high sodium oxybates have 1640 mg at the equivalent dose. Xywav is comprised of a unique composition of cations resulting in 92% less sodium, or a reduction of approximately 1,000 to 1,500 mg/night at the recommended dose range of 6 g to 9 g/night. Xywav is the only oxybate therapy that does not carry a warning in the label related to use in patients sensitive to high sodium intake.Xywav is also the first and only U.S. FDA-approved treatment option for idiopathic hypersomnia in adults. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of idiopathic hypersomnia in adults. Xywav is the only FDA-approved treatment studied across the multiple symptoms of idiopathic hypersomnia, such as EDS, sleep inertia (severe grogginess or confusion when waking up), long sleep duration and cognitive impairment. Xywav can be administered as a twice- or once-nightly regimen for the treatment of idiopathic hypersomnia in adults.The exact mechanism of action of Xywav in the treatment of adults with idiopathic hypersomnia and of cataplexy and EDS in narcolepsy is unknown. It is hypothesized that the therapeutic effects of Xywav are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as thalamocortical neurons.22 The U.S. Drug Enforcement Agency (DEA) has designated Xywav as a Schedule III medicine. The DEA defines Schedule III drugs, substances, or chemicals as drugs with a moderate to low potential for physical and psychological dependence.22,23 Because of the risks of central nervous system (CNS) depression and abuse and misuse, Xywav is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS.Important Safety Information for XywavWARNING: CENTRAL NERVOUS SYSTEM DEPRESSION and ABUSE AND MISUSE.Central Nervous System Depression
XYWAV is a CNS depressant. Clinically significant respiratory depression and obtundation may occur in patients treated with XYWAV at recommended doses. Many patients who received XYWAV during clinical trials in narcolepsy and idiopathic hypersomnia were receiving CNS stimulants.
Abuse and Misuse
The active moiety of XYWAV is oxybate or gamma-hydroxybutyrate (GHB). Abuse or misuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death.Because of the risks of CNS depression and abuse and misuse, XYWAV is available only through a restricted program under a REMS called the XYWAV and XYREM REMS.Contraindications
XYWAV is contraindicatedin combination with sedative hypnotics or alcohol andin patients with succinic semialdehyde dehydrogenase deficiency.Warnings and Precautions
Central Nervous System Depression
The concurrent use of XYWAV with other CNS depressants, including but not limited to opioid analgesics, benzodiazepines, sedating antidepressants or antipsychotics, sedating anti-epileptic drugs, general anesthetics, muscle relaxants, and/or illicit CNS depressants, may increase the risk of respiratory depression, hypotension, profound sedation, syncope, and death. If use of these CNS depressants in combination with XYWAV is required, dose reduction or discontinuation of one or more CNS depressants (including XYWAV) should be considered. In addition, if short-term use of an opioid (e.g., post- or perioperative) is required, interruption of treatment with XYWAV should be considered.After first initiating treatment and until certain that XYWAV does not affect them adversely (e.g., impair judgment, thinking, or motor skills), caution patients against hazardous activities requiring complete mental alertness or motor coordination such as operating hazardous machinery, including automobiles or airplanes. Also caution patients against these hazardous activities for at least 6 hours after taking XYWAV. Patients should be queried about CNS depression-related events upon initiation of XYWAV therapy and periodically thereafter.Abuse and Misuse
XYWAV is a Schedule Ill controlled substance. The active moiety of XYWAV is oxybate, also known as GHB, a Schedule I controlled substance. Abuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death. The rapid onset of sedation, coupled with the amnestic features of GHB particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (e.g., assault victim). Physicians should carefully evaluate patients for a history of drug abuse and follow such patients closely.XYWAV and XYREM REMS
Because of the risks of central nervous system depression and abuse and misuse, XYWAV is available only through a restricted distribution program called the XYWAV and XYREM REMS.Notable requirements of the XYWAV and XYREM REMS include the following:Healthcare Providers who prescribe XYWAV are specially certifiedXYWAV will be dispensed only by the central pharmacy that is specially certifiedXYWAV will be dispensed and shipped only to patients who are enrolled in the XYWAV and XYREM REMS with documentation of safe useFurther information is available at www.XYWAVXYREMREMS.com or 1-866-997-3688.Respiratory Depression and Sleep-Disordered Breathing
XYWAV may impair respiratory drive, especially in patients with compromised respiratory function. In overdoses of oxybate and with illicit use of GHB, life-threatening respiratory depression has been reported. Increased apnea and reduced oxygenation may occur with XYWAV administration in adult and pediatric patients. A significant increase in the number of central apneas and clinically significant oxygen desaturation may occur in patients with obstructive sleep apnea treated with XYWAV. Prescribers should be aware that sleep-related breathing disorders tend to be more prevalent in obese patients, in men, in postmenopausal women not on hormone replacement therapy, and among patients with narcolepsy.Depression and Suicidality
In Study 1, the randomized-withdrawal clinical trial in adult patients with narcolepsy (n=201), depression and depressed mood were reported in 3% and 4%, respectively, of patients treated with XYWAV. Two patients (1%) discontinued XYWAV because of depression. In most cases, no change in XYWAV treatment was required. In Study 2, the randomized-withdrawal clinical trial in adult patients with idiopathic hypersomnia (n=154), depression and depressed mood were reported in 1% and 3%, respectively, of patients treated with XYWAV. All patients continued XYWAV treatment.Two suicides and two attempted suicides occurred in adult clinical trials with oxybate (same active moiety as XYWAV). One patient experienced suicidal ideation and two patients reported depression in a pediatric clinical trial with oxybate. These events occurred in patients with and without previous histories of depressive disorders. The emergence of depression in patients treated with XYWAV requires careful and immediate evaluation. Monitor patients for the emergence of increased depressive symptoms and/or suicidality while taking XYWAV.Other Behavioral or Psychiatric Adverse Reactions
In Study 1, confusion and anxiety occurred in 1% and 5% of patients with narcolepsy treated with XYWAV, respectively. One patient experienced visual hallucinations and confusion after ingesting approximately 9 grams of XYWAV.In Study 2, confusion and anxiety occurred in 3% and 16% of patients with idiopathic hypersomnia, respectively. One patient experienced visual hallucinations, which led to discontinuation of XYWAV. Other neuropsychiatric reactions reported with oxybate (same active moiety as XYWAV) in adult or pediatric clinical trials and in the postmarketing setting include hallucinations, paranoia, psychosis, aggression, agitation, confusion and anxiety. The emergence or increase in the occurrence of behavioral or psychiatric events in patients taking XYWAV should be carefully monitored.Parasomnias
Parasomnias can occur in patients taking XYWAV.In Study 1 and Study 2, parasomnias, including sleepwalking, were reported in 6% and 5% of adult patients treated with XYWAV, respectively. In a clinical trial of XYREM (same active moiety as XYWAV) in adult patients with narcolepsy, five instances of sleepwalking with potential injury or significant injury were reported. Parasomnias, including sleepwalking, have been reported in a pediatric clinical trial with sodium oxybate (same active moiety as XYWAV) and in postmarketing experience with sodium oxybate.Episodes of sleepwalking should be fully evaluated and appropriate interventions considered.Most Common Adverse Reactions
The most common adverse reactions (occurring in ≥5% of XYWAV-treated patients in adult clinical trials in either narcolepsy or IH) were nausea, headache, dizziness, anxiety, insomnia, decreased appetite, hyperhidrosis, vomiting, diarrhea, dry mouth, parasomnia, somnolence, fatigue, and tremor. In the pediatric clinical trial with XYREM (same active moiety as XYWAV) that included pediatric patients 7 to 17 years of age with narcolepsy, the most common adverse reactions (≥5%) were nausea (20%), enuresis (19%), vomiting (18%), headache (17%), weight decreased (13%), decreased appetite (9%), dizziness (8%), and sleepwalking (6%). The overall adverse reaction profile of XYREM in the pediatric clinical trial was similar to that seen in the adult clinical trial program. The safety profile in pediatric patients with XYWAV is expected to be similar to that of adult patients treated with XYWAV and to that of pediatric patients treated with XYREM.Additional Adverse Reactions
Adverse reactions that occurred in 2-
US Market News
4週前
Jazz Pharmaceuticals Provides Update on Zepzelca® (lurbinectedin) Phase 3 LAGOON Trial in Second-Line Small Cell Lung CancerJune 12, 2026 8:00 AM
PR Newswire (US) For U.S. media and investors onlyDUBLIN, June 12, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced top-line results from the Phase 3 LAGOON trial, conducted by PharmaMar, evaluating Zepzelca® (lurbinectedin) in patients with relapsed (second-line) metastatic small cell lung cancer (SCLC). The trial did not meet its primary endpoint of overall survival (OS) evaluating Zepzelca as monotherapy or in combination with irinotecan compared to investigators' choice of topotecan or irinotecan. No new safety signals were identified with Zepzelca monotherapy or in combination with irinotecan, and the overall safety profiles of the investigational arms were consistent with the known safety profile of each agent."Relapsed SCLC is an aggressive cancer with a poor prognosis and patients continue to need treatment options, including in later lines of therapy. We thank the investigators, trial sites and patients who were involved in the LAGOON trial, along with our partner, PharmaMar," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "Zepzelca is an important treatment in SCLC and, based on the strength of the IMforte trial results, we believe its most beneficial use is in the first-line maintenance setting in combination with immunotherapy given the rapid progression of metastatic SCLC after first-line chemotherapy induction."The full U.S. approval of Zepzelca in 2025 is based on the Phase 3 IMforte trial, which evaluated Zepzelca in combination with atezolizumab as first-line maintenance treatment for patients with extensive-stage SCLC. In the IMforte trial, the Zepzelca and atezolizumab combination demonstrated a statistically significant improvement in the primary endpoints of OS and progression-free survival (PFS), as assessed by an independent review facility, compared to treatment with atezolizumab alone. The Zepzelca and atezolizumab combination reduced the risk of disease progression or death by 46% and the risk of death by 27%, compared to atezolizumab maintenance therapy alone. The LAGOON results are distinct from the IMforte trial and do not impact Zepzelca's approval in the first-line maintenance setting.The company has shared the LAGOON results with the FDA and will discuss next steps with the agency regarding its post-marketing requirements for the Zepzelca second-line indication.The study results do not impact the company's 2026 guidance.Key Results from the Phase 3 LAGOON Trial
The LAGOON trial included a broader patient population than the Phase 2 pivotal trial that supported the second-line accelerated approval, including patients with a history of CNS involvement. Efficacy of Zepzelca in the subset of patients without a history of CNS involvement was more comparable to the control arm, which performed better than historical precedent.Trial PopulationZepzelca monotherapy
Median OSZepzelca + irinotecan
Median OSControl
Median OSHR (95% CI)
Zepzelca vs ControlHR (95% CI)
Zepzelca+irinotecan vs ControlOverall8.7 (n=240)10.9 (n=242)10.7 (n=242)1.190 (0.959, 1.476)0.902 (0.729, 1.115)Without CNS metastases 9.6 (n=182)11.1 (n=189)10.7 (n=186)1.106 (0.875, 1.398)0.922 (0.729, 1.166)With CNS metastases7.1 (n=58)10.5 (n= 53)10.3 (n=56)1.791 (1.162, 2.760)1.107 (0.724, 1.692)The overall safety profile for Zepzelca was favorable relative to the control arm. Treatment-related adverse events (TRAE) were 78.5% with Zepzelca, 95% with Zepzelca + irinotecan, and 93.8% with the control arm. TRAEs Grade ≥ 3 were 35% with Zepzelca, 62.6% with Zepzelca + irinotecan, and 64.4% with the control arm.About the LAGOON Trial
LAGOON (NCT05153239) is a Phase 3, randomized (1:1:1), multicenter, open-label clinical trial with three arms: one arm to receive lurbinectedin 3.2 mg/m2 as monotherapy (the approved dose in the U.S.), the second arm to receive lurbinectedin 2.0 mg/m2 in combination with irinotecan 75 mg/m2, and the third arm to receive topotecan or irinotecan based on the investigators' choice. The trial was conducted in patients with SCLC, whose disease has progressed following prior platinum-containing chemotherapy with or without anti-PD-1 or anti-PD-L1 agents. The LAGOON trial enrolled 724 patients from more than 200 sites globally, including in the U.S., Canada and Europe. The trial is sponsored by Jazz's partner, PharmaMar. About Small Cell Lung Cancer
In the U.S., approximately 13 percent of lung cancers are small cell.1 Approximately 30,000 new cases of small cell lung cancer (SCLC) are reported in the U.S. each year.2 The risk for developing SCLC is much higher among current or former tobacco smokers; however, SCLC can also be caused by exposure to secondhand smoke, asbestos, some inhaled chemicals, radiation and air pollution. People with a family history of lung cancer may also be at a higher risk.3 SCLC is an aggressive form of lung cancer and it tends to spread quickly to other parts of the body including the brain, liver and bone.4,5 A large percentage of SCLC patients on treatment briefly achieve a response, although the cancer often returns and is usually more aggressive and resistant to regimens that were previously effective.6About Zepzelca® (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and potentially cell death.7In October 2025, the FDA approved Zepzelca in combination with atezolizumab or atezolizumab and hyaluronidase-tqjs, as maintenance treatment for adults with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab or atezolizumab and hyaluronidase-tqjs, carboplatin and etoposide.In June 2020, the FDA approved Zepzelca for the treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. This indication is approved under accelerated approval based on ORR and DOR.Important Safety Information for ZEPZELCAMyelosuppression
ZEPZELCA can cause severe and fatal myelosuppression including febrile neutropenia and sepsis, thrombocytopenia and anemia.Administer ZEPZELCA only to patients with baseline neutrophil count of at least 1,500 cells/mm3 and platelet count of at least 100,000/mm3. To reduce the risk of febrile neutropenia during treatment with ZEPZELCA in combination with atezolizumab or atezolizumab and hyaluronidase-tqjs, administer granulocyte colony-stimulating factor (G-CSF). Monitor blood counts including neutrophils, red blood cells and platelets prior to each ZEPZELCA administration. For neutrophil count less than 500 cells/mm3 or any value less than lower limit of normal, the use of G-CSF is recommended. Withhold, reduce the dose, or permanently discontinue ZEPZELCA based on severity.ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, primary prophylaxis of G-CSF was administered to 84% of patients. Based on laboratory values, decreased neutrophils occurred in 36%, including 18% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. The median time to onset of Grade 3 and 4 decreased neutrophil cells was 31 days and a median duration of 10 days. Febrile neutropenia occurred in 1.7%. Sepsis occurred in 1%. There were 7 fatal infections: pneumonia (n=3), sepsis (n=3), and febrile neutropenia (n=1).Based on laboratory values, decreased platelets occurred in 54%, including 15% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. The median time to onset of Grade 3 and 4 decreased platelet cells was 31 days and a median duration of 12 days.Based on laboratory values, decreased hemoglobin occurred in 51%, including 13% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. The median time to onset of Grade 3 and 4 decreased hemoglobin was 64 days and a median duration of 8 days.ZEPZELCA as a Single AgentIn clinical studies of 554 patients with advanced solid tumors receiving ZEPZELCA as a single agent, Grade 3 or 4 neutropenia occurred in 41% of patients, with a median time to onset of 15 days and a median duration of 7 days. Febrile neutropenia occurred in 7% of patients. Sepsis occurred in 2% of patients and was fatal in 1% (all cases occurred in patients with solid tumors other than SCLC). Grade 3 or 4 thrombocytopenia occurred in 10%, with a median time to onset of 10 days and a median duration of 7 days. Grade 3 or 4 anemia occurred in 17% of patients.Hepatotoxicity
ZEPZELCA can cause hepatotoxicity which may be severe.Monitor liver function tests prior to initiating ZEPZELCA and periodically during treatment as clinically indicated. Withhold, reduce the dose, or permanently discontinue ZEPZELCA based on severity.ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, based on laboratory values, increased alanine aminotransferase (ALT) occurred in 25%, including 3% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. Increased aspartate aminotransferase (AST) occurred in 24% including 3% Grade 3 or Grade 4. The median time to onset of Grade ≥3 elevation in transaminases was 52 days (range: 6 to 337).ZEPZELCA as a Single AgentIn clinical studies of 554 patients with advanced solid tumors receiving ZEPZELCA as a single agent, Grade 3 elevations of ALT and AST were observed in 6% and 3% of patients, respectively, and Grade 4 elevations of ALT and AST were observed in 0.4% and 0.5% of patients, respectively. The median time to onset of Grade ≥3 elevation in transaminases was 8 days (range: 3 to 49), with a median duration of 7 days.Extravasation Resulting in Tissue Necrosis
Extravasation of ZEPZELCA can cause skin and soft tissue injury, including necrosis requiring debridement. Consider use of a central venous catheter to reduce the risk of extravasation, particularly in patients with limited venous access. Monitor patients for signs and symptoms of extravasation during the ZEPZELCA infusion. If extravasation occurs, immediately discontinue the infusion, remove the infusion catheter, and monitor for signs and symptoms of tissue necrosis. The time to onset of necrosis after extravasation may vary.ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, extravasation resulting in skin necrosis occurred in one patient who received ZEPZELCA in combination with atezolizumab.Administer supportive care and consult with an appropriate medical specialist as needed for signs and symptoms of extravasation. Administer subsequent infusions at a site that was not affected by extravasation.Rhabdomyolysis
Rhabdomyolysis has been reported in patients treated with ZEPZELCA.Monitor creatine phosphokinase (CPK) prior to initiating ZEPZELCA and periodically during treatment as clinically indicated. Withhold or reduce the dose based on severity. ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, among 235 patients who had a creatine phosphokinase laboratory evaluation, increased creatine phosphokinase occurred in 9% who received ZEPZELCA in combination with atezolizumab.Embryo-Fetal Toxicity
ZEPZELCA can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise female patients of reproductive potential to use effective contraception during treatment with ZEPZELCA and for 7 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ZEPZELCA and for 4 months after the last dose.Lactation
There are no data on the presence of ZEPZELCA in human milk, however, because of the potential for serious adverse reactions from ZEPZELCA in breastfed children, advise women not to breastfeed during treatment with ZEPZELCA and for 2 weeks after the last dose.ADVERSE REACTIONSZEPZELCA with Intravenous AtezolizumabSerious adverse reactions occurred in 31% of patients receiving ZEPZELCA in combination with atezolizumab. Serious adverse reactions occurring in >2% were pneumonia (2.5%), respiratory tract infections (2.1%), dyspnea (2.1%), and decreased platelet count (2.1%). Fatal adverse reactions occurred in 5% of patients receiving ZEPZELCA with atezolizumab including pneumonia (3 patients), sepsis (3 patients), cardio-respiratory arrest (2 patients), myocardial infarction (2 patients), and febrile neutropenia (1 patient).The most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received ZEPZELCA with atezolizumab were decreased lymphocytes (55%), decreased platelets (54%), decreased hemoglobin (51%), decreased neutrophils (36%), nausea (36%), and fatigue/asthenia (32%).ZEPZELCA as a Single AgentSerious adverse reactions occurred in 34% of patients who received ZEPZELCA. Serious adverse reactions in ≥3% of patients included pneumonia, febrile neutropenia, neutropenia, respiratory tract infection, anemia, dyspnea, and thrombocytopenia.The most common adverse reactions, including laboratory abnormalities, (≥20%) are leukopenia (79%), lymphopenia (79%), fatigue (77%), anemia (74%), neutropenia (71%), increased creatinine (69%), increased alanine aminotransferase (66%), increased glucose (52%), thrombocytopenia (37%), nausea (37%), decreased appetite (33%), musculoskeletal pain (33%), decreased albumin (32%), constipation (31%), dyspnea (31%), decreased sodium (31%), increased aspartate aminotransferase (26%), vomiting (22%), decreased magnesium (22%), cough (20%), and diarrhea (20%).DRUG INTERACTIONS
Effect of CYP3A Inhibitors and InducersAvoid coadministration with a strong or a moderate CYP3A inhibitor (including grapefruit and Seville oranges) as this increases lurbinectedin systemic exposure which may increase the incidence and severity of adverse reactions to ZEPZELCA. If coadministration cannot be avoided, reduce the ZEPZELCA dose as appropriate.Avoid coadministration with a strong CYP3A inducer as it may decrease systemic exposure to lurbinectedin, which may decrease the efficacy of ZEPZELCA.GERIATRIC USEZEPZELCA with Intravenous AtezolizumabOf the 242 patients with ES-SCLC treated with ZEPZELCA and atezolizumab in IMforte, 124 (51%) patients were 65 years of age and older, while 29 (12%) patients were 75 years of age and older. No overall differences in effectiveness were observed between older and younger patients. There was no overall difference in the incidence of serious adverse reactions in patients ≥65 years of age and patients
US Market News
1月前
Jazz Pharmaceuticals to Present Comprehensive Data at SLEEP 2026 Highlighting Broad Treatment Effects of Xywav® (calcium, magnesium, potassium, and sodium oxybates) Oral Solution for People with Narcolepsy and Idiopathic HypersomniaJune 4, 2026 7:45 AM
PR Newswire (US) Twenty-one abstracts, including 11 late-breaking abstracts, underscore Jazz's unwavering commitment to advancing the treatment of narcolepsy and idiopathic hypersomniaFor U.S. media and investors onlyDUBLIN, June 4, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the company will present 21 abstracts, including 11 late-breaking abstracts, at SLEEP 2026, the 40th annual meeting of the Associated Professional Sleep Societies (APSS), taking place June 14-17, 2026, in Baltimore. The robust breadth of data reflects Jazz's continued focus on advancing treatment for rare and difficult-to-treat sleep disorders with new clinical research and real-world evidence evaluating Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution in patients with narcolepsy and idiopathic hypersomnia."People living with narcolepsy and idiopathic hypersomnia, while profoundly affected by symptoms, face challenges in diagnosis and navigating treatment considerations," said Jessa Alexander, Ph.D., neuroscience therapeutic area head, global medical and scientific affairs of Jazz Pharmaceuticals. "The research Jazz is unveiling at SLEEP 2026 is rooted in the day-to-day realities of patients, reflecting our commitment to conduct studies that mirror real-world experiences and treatment paradigms. This comprehensive approach allows our research to inform care decisions and address the full complexity of these conditions, spanning the 24-hour burden of sleep inertia, daytime symptoms and sleep as well as the meaningful implications of reducing long-term sodium intake."Key highlights at SLEEP 2026 include:An oral presentation featuring a post-hoc analysis of the DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) study, examining objective and subjective sleep inertia in participants with narcolepsy (type 1 and type 2) taking Xywav.Two poster presentations reporting post-hoc analyses of the DUET study exploring outcomes for idiopathic hypersomnia participants taking Xywav treatment, with one evaluating objective and patient-reported sleep inertia and the other exploring outcomes of Xywav treatment when stratified by individual ideal sleep duration.A poster presentation showcasing changes in hypersomnolence among participants with narcolepsy taking Xywav dosages >9 grams per night, compared to 9 grams at baseline, in the DUET study. The current recommended dosage of Xywav for adults with narcolepsy is 6-9 grams per night.Six poster presentations featuring comprehensive findings from the real-world, longitudinal, mixed-methods LYRICAL study, examining the treatment experience and dosing patterns of U.S. adults with idiopathic hypersomnia or narcolepsy taking Xywav.Two poster presentations detailing the development of conceptual disease models of the symptoms and impact of narcolepsy type 1 and narcolepsy type 2, respectively, from the patient perspective to better understand the most bothersome symptoms and meaningful impacts.The SLEEP 2026 abstracts are available online at: sleepmeeting.org/abstract-supplements.The full list of Jazz's presentations at SLEEP 2026 is:Presentation TitleAuthorsPresentation DetailsImprovements in Sleep Inertia With
Low-Sodium Oxybate Treatment in
Participants With Narcolepsy Type
1 and Type 2 in the DUET StudyNichols DA, Schneider LD,
Plante DT, Dai J, Steininger
TL, Whalen M, Cairns A,
Ruoff CM, Van Dongen HPAType: Oral and poster Oral Session: O-23Oral Date/Time: June 17, 2026, 4:45-5:00 p.m. ETPoster Session: P-35Poster Date/Time: June 16, 2026,10:00-11:45 a.m. ETPoster Board Number: 339Clinical Responders to Low-Sodium
Oxybate in Narcolepsy and
Idiopathic HypersomniaRuoff CM, Sangal RB,
Nichols DA, Steininger TL,
Dai J, Vahabzadeh
S, Whalen W, Markt SC,
Herpel LB, Foldvary-
Schaefer NType: Late-breaking oral and poster Oral Session: LBA-02Oral Date/Time: June 16, 2026, 11:00-11:15 a.m. ETPoster Session: P-41Poster Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 421Estimating the Prevalence of
Diagnosed Idiopathic Hypersomnia
in the Pediatric Population: A US
Claims AnalysisGavrielov-Yusim N, Markt
SC, Nelms J, Bhattacharjee
R, Maski KType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 539Clinically Relevant Symptoms of
Narcolepsy and Idiopathic
Hypersomnia in Patients Initiating
Low-Sodium Oxybate: Electronic
Health Record Notes StudyMarkt SC, Whalen M,
Drachenberg C, Beaty S,
Alexander JK, Casstevens
C, Fee RM, Russell K, Ortiz
LEType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 533Improvements in Nighttime Sleep
Quality and Daytime Sleepiness
Are Associated With Better Quality
of Life in Narcolepsy: Findings
From LYRICALDrachenberg C, Farrell M,
D'Souza J, Kim E, Hayes
C, Zhang D, Lawrence M,
Graham CA, Whalen M,
Alexander JK, Beaty S,
Markt SC, Herpel LBType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 534Daytime and Nighttime Symptom
Improvements Are Associated With
Better Quality of Life in Idiopathic
Hypersomnia: Findings From
LYRICALDrachenberg C, Farrell
M, D'Souza J, Kim E,
Hayes C, Zhang D,
Lawrence M, Graham CA,
Whalen M, Alexander JK,
Beaty S, Markt SC, Herpel
LBType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 535Changes in Cardiovascular and
Cardiometabolic Biomarkers
Following Low-Sodium Oxybate
Initiation for Narcolepsy or
Idiopathic HypersomniaMarkt SC, Alexander JK,
Hughes AG, Sachdev A,
Whalen M, Drachenberg C,
Beaty S, Dai J, Black J,
Somers VKType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 526Nocturnal Sleep Features as a
Candidate Mechanism for
Improvements in Daytime
Symptoms in Idiopathic
Hypersomnia or NarcolepsyCairns A, Plante DT, Ruoff
CM, Schneider LD, Dai J,
Nichols DA, Steininger TL,
Whalen M, Markt SC,
Bogan RK, Dauvilliers Y,
Mignot EType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 537Nocturnal Sleep Features as
Candidates for Evaluating Disease
Specific Improvements in Idiopathic
Hypersomnia or NarcolepsyCairns A, Plante DT, Ruoff
CM, Schneider LD, Dai J,
Nichols DA, Steininger TL,
Whalen M, Markt SC,
Bogan RK, Dauvilliers Y,
Mignot EType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 536Changes in Patient-Reported
Symptoms After Switching From
High- to Low-Sodium Oxybate in
Participants With Narcolepsy in the
XYLO StudySomers VK, Dauvilliers Y,
Nichols DA, Markt SC,
Baranak C, Dai J, Measey
TJ, Whalen M, White WBType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 527Individualized Dosing and Drug
Utilization of Low-Sodium Oxybate
in Narcolepsy and Idiopathic
Hypersomnia: Results from Post-
Marketing DataGavrielov-Yusim N, Nelms
J, Dai J, Balmuri P,
Saumweber A, Singer D,
Skobieranda F, Patodiya V,
Markt SType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 529Individualized Dosing of Low-
Sodium Oxybate in Patients with
Narcolepsy or Idiopathic
HypersomniaFoldvary-Schaefer N,
Meskill GJ, Nichols DA,
Steininger TL, Dai J,
Measey T, Whalen M,
Cairns A, Schneider LD,
Bogan RKType: Late-breaking posterSession: P-41Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 528Improvements in Sleep Inertia With
Low-Sodium Oxybate Treatment in
Participants With Idiopathic
Hypersomnia in the DUET StudyNichols DA, Schneider LD,
Ruoff CM, Dai J, Steininger
TL, Whalen M, Cairns A,
Plante DT, Van Dongen
HPAType: PosterSession: P-35Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 338Patient-Reported Improvement in
Hypersomnolence Measures in
Participants With Narcolepsy
Taking >9 Gram Dosage of Low-
Sodium Oxybate in the DUET StudyBogan RK, Ruoff CM,
Plante DT, Nichols DA,
Steininger TL, Dai J,
Measey TJ, Whalen M,
Cairns A, Schneider LD,
Simmons JHType: PosterSession: P-35Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 331Effectiveness and Safety of Low-
Sodium Oxybate by Patient-
Reported Long Sleep Need in
DUET Study Participants With
Idiopathic HypersomniaPlante DT, Ruoff CM,
Schneider LD, Nichols DA,
Steininger TL, Cairns A, Dai
J, Whalen M, Dauvilliers YType: PosterSession: P-35Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 325A Conceptual Disease Model of the
Symptoms and Impacts of
Narcolepsy Type 1 From the
Patient PerspectiveCasstevens C, Foldvary-
Schaefer N, Maski K,
Plante DT, Farrell M,
Graham CA, Mobley C, Kim
E, Wraight M, Black J,
Steinerman JR, Ortiz LEType: PosterSession: P-34Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 302A Conceptual Disease Model of the
Symptoms and Impacts of
Narcolepsy Type 2 From the
Patient PerspectiveCasstevens C, Maski K,
Foldvary-Schaefer N, Ortiz
LE, Farrell M, Graham CA,
Mobley C, Kim E, Wraight
M, Black J, Steinerman JR,
Plante DTType: PosterSession: P-34Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 303Effectiveness and Satisfaction With
Low-Sodium Oxybate As Described
by Adults Living With Narcolepsy:
LYRICAL Survey & Interview
FindingsDrachenberg C, Farrell M,
D'Souza J, Kim E,
Lawrence M, Graham CA,
Whalen M, Alexander JK,
Beaty S, Markt SC, Herpel
LBType: PosterSession: P-38Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 359Effectiveness and Satisfaction With
Low-Sodium Oxybate As Described
by Adults Living With Idiopathic
Hypersomnia: LYRICAL Survey &
Interview FindingsDrachenberg C, Farrell M,
D'Souza J, Kim E,
Lawrence M, Graham CA,
Whalen M, Alexander JK,
Beaty S, Markt SC, Herpel
LBType: PosterSession: P-38Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 360Individualized Dosing With Low-
Sodium Oxybate in Narcolepsy:
Patient Voices From LYRICALDrachenberg C, Farrell M,
D'Souza J, Kim E,
Lawrence M, Graham CA,
Whalen M, Alexander JK,
Beaty S, Markt SC, Herpel
LBType: PosterSession: P-38Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 362Individualized Dosing With Low-
Sodium Oxybate in Idiopathic
Hypersomnia: Patient Voices From
LYRICALDrachenberg C, Farrell M,
D'Souza J, Kim E,
Lawrence M, Graham CA,
Whalen M, Alexander JK,
Beaty S, Markt SC, Herpel
LBType: PosterSession: P-38Date/Time: June 16, 2026, 10:00-11:45 a.m. ETPoster Board Number: 361About Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution
Xywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy. The Office of Orphan Product Development (OOPD) at the FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy by means of greater cardiovascular safety compared to Xyrem® (sodium oxybate) oral solution. The decision of the OOPD is based on the FDA findings that Xywav provides a greatly reduced chronic sodium burden compared to Xyrem. Xywav has 131 mg of sodium at the maximum recommended nightly dose whereas other high sodium oxybates have 1640 mg at the equivalent dose. Xywav is comprised of a unique composition of cations resulting in 92% less sodium, or a reduction of approximately 1,000 to 1,500 mg/night at the recommended dose range of 6 g to 9 g/night. Xywav is the only oxybate therapy that does not carry a warning in the label related to use in patients sensitive to high sodium intake.Xywav is also the first and only FDA-approved treatment option for idiopathic hypersomnia in adults. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of idiopathic hypersomnia in adults. Xywav is the only FDA-approved treatment studied across the multiple symptoms of idiopathic hypersomnia, such as EDS, sleep inertia (severe grogginess or confusion when waking up), long sleep duration and cognitive impairment. Xywav can be administered as a twice- or once-nightly regimen for the treatment of idiopathic hypersomnia in adults.The exact mechanism of action of Xywav in the treatment of adults with idiopathic hypersomnia and of cataplexy and EDS in narcolepsy is unknown. It is hypothesized that the therapeutic effects of Xywav are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as thalamocortical neurons.1 The U.S. Drug Enforcement Agency (DEA) has designated Xywav as a Schedule III medicine. The DEA defines Schedule III drugs, substances, or chemicals as drugs with a moderate to low potential for physical and psychological dependence.1,2 Because of the risks of central nervous system (CNS) depression and abuse and misuse, Xywav is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS.Important Safety Information for XywavWARNING: CENTRAL NERVOUS SYSTEM DEPRESSION and ABUSE AND MISUSE.Central Nervous System Depression
XYWAV is a CNS depressant. Clinically significant respiratory depression and obtundation may occur in patients treated with XYWAV at recommended doses. Many patients who received XYWAV during clinical trials in narcolepsy and idiopathic hypersomnia were receiving CNS stimulants.
Abuse and Misuse
The active moiety of XYWAV is oxybate or gamma-hydroxybutyrate (GHB). Abuse or misuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death.Because of the risks of CNS depression and abuse and misuse, XYWAV is available only through a restricted program under a REMS called the XYWAV and XYREM REMS.Contraindications
XYWAV is contraindicatedin combination with sedative hypnotics or alcohol andin patients with succinic semialdehyde dehydrogenase deficiency.Warnings and Precautions
Central Nervous System Depression
The concurrent use of XYWAV with other CNS depressants, including but not limited to opioid analgesics, benzodiazepines, sedating antidepressants or antipsychotics, sedating anti-epileptic drugs, general anesthetics, muscle relaxants, and/or illicit CNS depressants, may increase the risk of respiratory depression, hypotension, profound sedation, syncope, and death. If use of these CNS depressants in combination with XYWAV is required, dose reduction or discontinuation of one or more CNS depressants (including XYWAV) should be considered. In addition, if short-term use of an opioid (e.g., post- or perioperative) is required, interruption of treatment with XYWAV should be considered.After first initiating treatment and until certain that XYWAV does not affect them adversely (e.g., impair judgment, thinking, or motor skills), caution patients against hazardous activities requiring complete mental alertness or motor coordination such as operating hazardous machinery, including automobiles or airplanes. Also caution patients against these hazardous activities for at least 6 hours after taking XYWAV. Patients should be queried about CNS depression-related events upon initiation of XYWAV therapy and periodically thereafter.Abuse and Misuse
XYWAV is a Schedule Ill controlled substance. The active moiety of XYWAV is oxybate, also known as GHB, a Schedule I controlled substance. Abuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death. The rapid onset of sedation, coupled with the amnestic features of GHB particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (e.g., assault victim). Physicians should carefully evaluate patients for a history of drug abuse and follow such patients closely.XYWAV and XYREM REMS
Because of the risks of central nervous system depression and abuse and misuse, XYWAV is available only through a restricted distribution program called the XYWAV and XYREM REMS.Notable requirements of the XYWAV and XYREM REMS include the following:Healthcare Providers who prescribe XYWAV are specially certifiedXYWAV will be dispensed only by the central pharmacy that is specially certifiedXYWAV will be dispensed and shipped only to patients who are enrolled in the XYWAV and XYREM REMS with documentation of safe useFurther information is available at www.XYWAVXYREMREMS.com or 1-866-997-3688.Respiratory Depression and Sleep-Disordered Breathing
XYWAV may impair respiratory drive, especially in patients with compromised respiratory function. In overdoses of oxybate and with illicit use of GHB, life-threatening respiratory depression has been reported. Increased apnea and reduced oxygenation may occur with XYWAV administration in adult and pediatric patients. A significant increase in the number of central apneas and clinically significant oxygen desaturation may occur in patients with obstructive sleep apnea treated with XYWAV. Prescribers should be aware that sleep-related breathing disorders tend to be more prevalent in obese patients, in men, in postmenopausal women not on hormone replacement therapy, and among patients with narcolepsy.Depression and Suicidality
In Study 1, the randomized-withdrawal clinical trial in adult patients with narcolepsy (n=201), depression and depressed mood were reported in 3% and 4%, respectively, of patients treated with XYWAV. Two patients (1%) discontinued XYWAV because of depression. In most cases, no change in XYWAV treatment was required. In Study 2, the randomized-withdrawal clinical trial in adult patients with idiopathic hypersomnia (n=154), depression and depressed mood were reported in 1% and 3%, respectively, of patients treated with XYWAV. All patients continued XYWAV treatment.Two suicides and two attempted suicides occurred in adult clinical trials with oxybate (same active moiety as XYWAV). One patient experienced suicidal ideation and two patients reported depression in a pediatric clinical trial with oxybate. These events occurred in patients with and without previous histories of depressive disorders. The emergence of depression in patients treated with XYWAV requires careful and immediate evaluation. Monitor patients for the emergence of increased depressive symptoms and/or suicidality while taking XYWAV.Other Behavioral or Psychiatric Adverse Reactions
In Study 1, confusion and anxiety occurred in 1% and 5% of patients with narcolepsy treated with XYWAV, respectively. One patient experienced visual hallucinations and confusion after ingesting approximately 9 grams of XYWAV.In Study 2, confusion and anxiety occurred in 3% and 16% of patients with idiopathic hypersomnia, respectively. One patient experienced visual hallucinations, which led to discontinuation of XYWAV. Other neuropsychiatric reactions reported with oxybate (same active moiety as XYWAV) in adult or pediatric clinical trials and in the postmarketing setting include hallucinations, paranoia, psychosis, aggression, agitation, confusion and anxiety. The emergence or increase in the occurrence of behavioral or psychiatric events in patients taking XYWAV should be carefully monitored.Parasomnias
Parasomnias can occur in patients taking XYWAV.In Study 1 and Study 2, parasomnias, including sleepwalking, were reported in 6% and 5% of adult patients treated with XYWAV, respectively. In a clinical trial of XYREM (same active moiety as XYWAV) in adult patients with narcolepsy, five instances of sleepwalking with potential injury or significant injury were reported. Parasomnias, including sleepwalking, have been reported in a pediatric clinical trial with sodium oxybate (same active moiety as XYWAV) and in postmarketing experience with sodium oxybate.Episodes of sleepwalking should be fully evaluated and appropriate interventions considered.Most Common Adverse Reactions
The most common adverse reactions (occurring in ≥5% of XYWAV-treated patients in adult clinical trials in either narcolepsy or IH) were nausea, headache, dizziness, anxiety, insomnia, decreased appetite, hyperhidrosis, vomiting, diarrhea, dry mouth, parasomnia, somnolence, fatigue, and tremor. In the pediatric clinical trial with XYREM (same active moiety as XYWAV) that included pediatric patients 7 to 17 years of age with narcolepsy, the most common adverse reactions (≥5%) were nausea (20%), enuresis (19%), vomiting (18%), headache (17%), weight decreased (13%), decreased appetite (9%), dizziness (8%), and sleepwalking (6%). The overall adverse reaction profile of XYREM in the pediatric clinical trial was similar to that seen in the adult clinical trial program. The safety profile in pediatric patients with XYWAV is expected to be similar to that of adult patients treated with XYWAV and to that of pediatric patients treated with XYREM.Additional Adverse Reactions
Adverse reactions that occurred in 2-
US Market News
2月前
Jazz Pharmaceuticals Announces First Quarter 2026 Financial ResultsMay 5, 2026 4:05 PM
PR Newswire (US) – Strong commercial execution across franchises with total revenues of $1.1 billion (+19% YoY) –– Zanidatamab HER2+ 1L GEA sBLA granted Priority Review; PDUFA date of August 25, 2026 –– Xywav® revenues grew 18% YoY with 425 net patient adds –– Epidiolex® revenues grew 15% YoY–– Company reaffirms 2026 revenue and expense guidance –DUBLIN, May 5, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced financial results for the first quarter of 2026 (1Q26)."Our first-quarter results reflect disciplined execution across the business, delivering 19% year-over-year growth alongside key pipeline advancements and positioning the company for an outstanding 2026," said Renee Gala, president and chief executive officer of Jazz Pharmaceuticals. "Demand for Xywav remained strong, our rare oncology launches with Modeyso™ and Zepzelca® in 1LM ES-SCLC gained significant momentum, and Epidiolex continued to provide consistent growth. Looking ahead, we are excited about the potential launch of zanidatamab in 1L GEA later this year, as we progress our pipeline and business development efforts to bring more life-changing therapies to patients and fuel durable long-term growth."Key First Quarter 2026 HighlightsTotal revenues in 1Q26 grew to $1.1 billion (+19% year-over-year (YoY))Generated GAAP / non-GAAP1 adjusted earnings per share (EPS) of $4.43 / $6.34 with $408 million in cash from operations.Practice-changing Phase 3 HERIZON-GEA-01 results, presented as a late-breaker at ASCO GI, support zanidatamab as the HER2-targeted agent of choice in HER2+ 1L advanced gastroesophageal adenocarcinoma (GEA); additional benefit from tislelizumab irrespective of PD-L1 status.Supplemental Biologics License Application (sBLA) accepted by FDA under Real-Time Oncology Review (RTOR) program with potential approval and launch in 1L HER2+ GEA on or before PDUFA date.Business UpdatesXywav (calcium, magnesium, potassium, and sodium oxybates) oral solution:Xywav net product sales increased 18% to $408 million in 1Q26, compared to 1Q25.Continued physician and patient demand for the differentiated benefits of low-sodium Xywav.Strong new patient growth, with approximately 425 net patient adds in 1Q26. There were approximately 16,600 active patients exiting the quarter, comprised of approximately 11,075 narcolepsy patients and approximately 5,525 idiopathic hypersomnia (IH) patients.________________________________1 Non-GAAP adjusted EPS is a non-GAAP financial measure. See 'Non-GAAP Financial Measures' below for more information. A reconciliation of GAAP reported EPS to non-GAAP adjusted EPS is included at the end of this press release.Epidiolex/Epidyolex (cannabidiol):Epidiolex/Epidyolex net product sales increased 15% YoY to $250 million in 1Q26, driven by continued strong demand.Announced agreement with Nippon Zoki to commercialize Epidyolex in Japan, following completion of ongoing clinical trials and potential regulatory approval.Ziihera® (zanidatamab-hrii):Ziihera net product sales in biliary tract cancer (BTC) were $13 million in 1Q26.In April 2026, FDA accepted the zanidatamab sBLA in GEA for a Priority Review, with a PDUFA date of August 25, 2026.Submitted HERIZON-GEA-01 data for potential National Comprehensive Cancer Network (NCCN) guideline inclusion.HERIZON-GEA-01 data accepted for publication by a top-tier medical journal.The second interim overall survival (OS) analysis for the HERIZON-GEA-01 trial doublet regimen is expected in mid-2026.Multiple registrational trials of zanidatamab are underway, including in metastatic breast cancer (mBC), supporting a broad development program designed to maximize patient impact and long-term shareholder value.Modeyso (dordaviprone):Modeyso net product sales were $41 million in 1Q26 with ~500 patients having received Modeyso from product launch in August 2025 through the end of the first quarter.The company completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $200 million (50% to Jazz).Phase 3 ACTION trial remains on track with top-line readout expected late 2026 / early 2027.Zepzelca (lurbinectedin):Zepzelca net product sales increased 60% YoY to $101 million in 1Q26, driven by continued uptake of the Zepzelca and atezolizumab combination in the 1LM ES-SCLC setting, partially offset by a decline in second line use.The company expects second line use to decline throughout the year.Financial Highlights
Three Months EndedMarch 31,(In millions, except per share amounts)2026
2025Total revenues$ 1,068.9
$ 897.8GAAP net income (loss)$ 293.1
$ (92.5)Non-GAAP adjusted net income$ 419.5
$ 105.2GAAP earnings (loss) per share$ 4.43
$ (1.52)Non-GAAP adjusted earnings per share$ 6.34
$ 1.68The GAAP net loss and non-GAAP adjusted net income for 1Q25 included an expense of $172 million related to Xyrem antitrust litigation settlements, which impacted our GAAP and non-GAAP results by $146 million (net of tax of $26 million), or $2.38 per share on a GAAP basis and $2.34 per share on a non-GAAP adjusted basis.Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.Total Revenues
Three Months EndedMarch 31,(In millions)2026
2025Xywav$ 408.2
$ 344.8Xyrem31.2
37.2Sleep439.4
382.0Epidiolex/Epidyolex249.8
217.7Epilepsy249.8
217.7Rylaze/Enrylaze103.7
94.2Zepzelca101.0
63.0Defitelio/defibrotide 47.4
40.7Modeyso41.4
—Vyxeos26.6
29.5Ziihera13.3
2.0Oncology333.4
229.4Other2.7
10.3Product sales, net1,025.3
839.4High-sodium oxybate AG royalty revenue36.3
48.9Other royalty and contract revenues7.3
9.5Total revenues$ 1,068.9
$ 897.8Total revenues increased 19% YoY primarily due to higher Xywav, Zepzelca and Epidiolex/Epidyolex net product sales and the inclusion of Modeyso net product sales, following FDA approval in August 2025.Operating Expenses and Income Tax Expense (Benefit)
Three Months EndedMarch 31,(In millions, except percentages)2026
2025GAAP:
Cost of product sales$ 134.1
$ 104.6Gross margin on total revenues87.5 %
88.3 %Selling, general and administrative$ 352.7
$ 514.0% of total revenues33.0 %
57.3 %Research and development$ 196.0
$ 180.7% of total revenues18.3 %
20.1 %Gain on sale of priority review voucher$ (122.8)
$ —Income tax expense (benefit) $ 6.1
$ (17.8)Effective tax rate2.0 %
16.2 %
Three Months EndedMarch 31,(In millions, except percentages)2026
2025Non-GAAP adjusted:
Cost of product sales$ 90.0
$ 69.7Gross margin on total revenues 91.6 %
92.2 %Selling, general and administrative$ 308.5
$ 472.3% of total revenues28.9 %
52.6 %Research and development$ 172.3
$ 159.7% of total revenues16.1 %
17.8 %Income tax expense$ 41.2
$ 36.5Effective tax rate8.9 %
25.7 %Changes in operating expenses and income tax expense (benefit) in 1Q26 over the prior year period are primarily due to the following:Cost of product sales, on a GAAP and non-GAAP adjusted basis, increased in 1Q26, primarily due to higher royalty expenses, driven by higher revenues, and increased inventory provisions. The cost of product sales, on a GAAP basis, in 1Q26 included higher acquisition accounting inventory fair value step up expense compared to 1Q25.Selling, general and administrative (SG&A) expenses, on a GAAP and non-GAAP adjusted basis, decreased in 1Q26, primarily due to certain Xyrem antitrust litigation settlements of $172 million incurred in 1Q25, partially offset by higher compensation-related expenses in 1Q26 including costs relating to Modeyso.Research and development (R&D) expenses, on a GAAP and non-GAAP adjusted basis, increased in 1Q26, primarily due to the addition of costs relating to Modeyso including personnel costs.Income tax expense in 1Q26, on a GAAP basis, was primarily attributable to the gain recognized on the sale of the PRV, partially offset by excess tax benefits from share-based compensation. Income tax benefit in 1Q25, on a GAAP basis, was primarily attributable to the Xyrem antitrust litigation settlements. Cash Flow and Balance Sheet As of March 31, 2026, cash, cash equivalents and investments were $2.9 billion, and the outstanding principal balance of the company's long-term debt was $5.4 billion. In addition, the company had undrawn borrowing capacity under a revolving credit facility of $885 million. For the three months ended March 31, 2026, the company generated $408 million of cash from operations reflecting strong business performance and continued financial discipline. In 1Q26, the company received gross proceeds of $200 million (50% to Jazz) from the sale of the PRV.2026 Financial Guidance (In millions)
GuidanceTotal Revenues
$4,250 - $4,500
(In millions, except percentages)GAAP
Non-GAAPGross margin %89% - 90%
90% - 91%1SG&A expenses$1,424 - $1,497
$1,260 - $1,3201R&D expenses$811 - $867
$725 - $7751Effective tax rate0% - 10%
11.5% - 13.5%1Weighted-average ordinary shares outstanding266 - 67
66 - 67___________________________1.See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included in the table titled "Reconciliation of 2026 GAAP to Non-GAAP Guidance Measures".2.Prior guidance as of February 24, 2026 was 65-66 million weighted-average ordinary shares outstanding. Guidance assumes inclusion of shares outstanding in relation to the 2.000% exchangeable senior notes due 2026 and the 3.125% exchangeable senior notes due 2030, which we refer to collectively as the Exchangeable Senior Notes, given the company's share price exceeds the conversion prices of the Exchangeable Senior Notes.Conference Call DetailsJazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET to provide a business and financial update and discuss its 2026 first quarter results.Interested parties may register for the call here or via the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast.A replay of the webcast will be available via the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com.About Jazz PharmaceuticalsJazz Pharmaceuticals plc (NASDAQ: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with rare disease — often with limited or no therapeutic options. We have a diverse portfolio of medicines, including leading therapies addressing epilepsies, cancers and sleep disorders. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information.Non-GAAP Financial MeasuresTo supplement Jazz Pharmaceuticals' financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the company uses certain non-GAAP (also referred to as adjusted or non-GAAP adjusted) financial measures in this press release and the accompanying tables. In particular, the company presents non-GAAP adjusted net income (and the related per share measure) and its line-item components, as well as certain non-GAAP adjusted financial measures derived therefrom, including non-GAAP adjusted gross margin percentage and non-GAAP adjusted effective tax rate. Non-GAAP adjusted net income (and the related per share measure) and its line-item components exclude from GAAP reported net income (loss) (and the related per share measure) and its line-item components certain items, as detailed in the reconciliation tables that follow, and in the case of non-GAAP adjusted net income (and the related per share measure), adjust for the income tax effect of the non-GAAP adjustments. In this regard, the components of non-GAAP adjusted net income, including non-GAAP adjusted cost of product sales, SG&A expenses and R&D expenses, are income statement line items prepared on the same basis as, and therefore components of, the overall non-GAAP adjusted net income measure.The company believes that each of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors and analysts and that each of these non-GAAP financial measures, when considered together with the company's financial information prepared in accordance with GAAP, can enhance investors' and analysts' ability to meaningfully compare the company's results from period to period, to its forward-looking guidance, and to identify operating trends in the company's business. In addition, these non-GAAP financial measures are regularly used by investors and analysts to model and track the company's financial performance. Jazz Pharmaceuticals' management also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate the company's business and to make operating decisions, and compensation of executives is based in part on certain of these non-GAAP financial measures. Because these non-GAAP financial measures are important internal measurements for Jazz Pharmaceuticals' management, the company also believes that these non-GAAP financial measures are useful to investors and analysts since these measures allow for greater transparency with respect to key financial metrics the company uses in assessing its own operating performance and making operating decisions. These non-GAAP financial measures are not meant to be considered in isolation or as a substitute for comparable GAAP measures; should be read in conjunction with the company's consolidated financial statements prepared in accordance with GAAP; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles in the reconciliation tables that follow. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its non-GAAP financial measures; and the company has ceased, and may in the future cease, to exclude items that it has historically excluded for purposes of its non-GAAP financial measures. Likewise, the company may determine to modify the nature of its adjustments to arrive at its non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measures as used by Jazz Pharmaceuticals in this press release and the accompanying tables have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.Cautionary Note Concerning Forward-Looking StatementsThis press release contains forward-looking statements, including, but not limited to, statements related to: the company's growth prospects and future financial and operating results, including the company's 2026 financial guidance and the company's expectations related thereto, including with respect to anticipated catalysts; the company's advancement of pipeline programs and the timing of development activities, regulatory activities, approvals, and submissions related thereto; the potential for a near-term commercial launch of zanidatamab in 1L HER2+ GEA in the U.S., if approved; planned or anticipated clinical trial events, including with respect to initiations, enrollment and data read-outs, and the anticipated timing thereof, including: the second interim OS data from the Phase 3 HERIZON trial of zanidatamab in 1L GEA and top-line data from the Phase 3 ACTION trial of Modeyso in recurrent H3 K27M-mutant diffuse glioma; and the company's development, regulatory and commercialization strategy; the company's expectations with respect to its products and product candidates and the potential of the company's products and product candidates and the potential regulatory path related thereto, including zanidatamab's potential to become the HER2-targeted therapy of choice in 1L HER2+ GEA, regardless of PD-L1 status; the company's capital allocation and corporate development strategy; the potential successful future development, manufacturing, regulatory and commercialization activities; the company's ability to realize the commercial potential of its products; the company's net product sales and goals for net product sales from new and acquired products; the company's views and expectations relating to its patent portfolio, including with respect to expected patent protection, as well as expectations with respect to exclusivity; the company's clinical trials confirming clinical benefit or enabling regulatory submissions, including the potential of the ongoing Phase 3 ACTION trial to confirm clinical benefit of Modeyso in recurrent H3 K27M-mutant diffuse glioma and extend to use in 1L patients; and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties.Actual results and the timing of events could differ materially from those anticipated in such forward- looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: maintaining or increasing sales of, and revenue from, Xywav, Epidiolex/Epidyolex, Ziihera, Modeyso, Zepzelca and other lead marketed products; effectively launching and commercializing the company's other products and product candidates; the successful completion of development and regulatory activities with respect to the company's product candidates; obtaining and maintaining adequate coverage and reimbursement for the company's products; the time-consuming and uncertain regulatory approval process, including the risk that the company's current and/or planned regulatory submissions may not be submitted, accepted or approved by applicable regulatory authorities in a timely manner or at all, including the risk that zanidatamab in 1L HER2+ GEA may not be approved in a timely manner or at all; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to the company's business operations and financial results; protecting and enhancing the company's intellectual property rights and the company's commercial success being dependent upon the company obtaining, maintaining and defending intellectual property protection and exclusivity for its products and product candidates; delays or problems in the supply or manufacture of the company's products and product candidates, including due to geopolitical tensions and military conflicts; complying with applicable U.S. and non-U.S. regulatory requirements, including those governing the research, development, manufacturing and distribution of controlled substances; government investigations, legal proceedings and other actions; identifying and consummating corporate development transactions, financing these transactions and successfully integrating acquired products, product candidates and businesses; the company's ability to realize the anticipated benefits of its collaborations and license agreements with third parties; the sufficiency of the company's cash flows and capital resources; the company's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the company's ability to meet its projected long-term goals and objectives, in the time periods that the company anticipates, or at all, and the inherent uncertainty and significant judgments and assumptions underlying the company's long-term goals and objectives; fluctuations in the market price and trading volume of the company's ordinary shares; and other risks and uncertainties affecting the company, including those described from time to time under the caption "Risk Factors" and elsewhere in the company's Securities and Exchange Commission filings and reports, including the company's Annual Report on Form 10-K for the year ended December 31, 2025 and future filings and reports by the company. Other risks and uncertainties of which the company is not currently aware may also affect the company's forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated.JAZZ PHARMACEUTICALS PLCCONDENSED CONSOLIDATED STATEMENTS OF INCOME (LOSS)(In millions, except per share amounts)(Unaudited)
Three Months EndedMarch 31,
2026
2025Revenues:
Product sales, net$ 1,025.3
$ 839.4Royalties and contract revenues43.6
58.4Total revenues1,068.9
897.8Operating expenses:
Cost of product sales (excluding amortization of acquired developed technologies)134.1
104.6Selling, general and administrative352.7
514.0Research and development196.0
180.7Intangible asset amortization172.3
154.4Gain on sale of priority review voucher(122.8)
—Total operating expenses732.3
953.7Income (loss) from operations336.6
(55.9)Interest expense, net(39.9)
(53.7)Foreign exchange gain (loss)2.5
(0.2)Income (loss) before income tax expense (benefit) and equity in loss of investees299.2
(109.8)Income tax expense (benefit) 6.1
(17.8)Equity in loss of investees—
0.5Net income (loss)$ 293.1
$ (92.5)
Net income (loss) per ordinary share:
Basic $ 4.73
$ (1.52)Diluted$ 4.43
$ (1.52)Weighted-average ordinary shares used in per share calculations - basic61.9
61.0Weighted-average ordinary shares used in per share calculations - diluted66.1
61.0 JAZZ PHARMACEUTICALS PLCCONDENSED CONSOLIDATED BALANCE SHEETS(In millions)(Unaudited)
March 31,
2026
December 31,
2025ASSETS
Current assets:
Cash and cash equivalents$ 1,844.3
$ 1,391.9Investments1,030.0
1,050.0Accounts receivable, net of allowances836.3
830.7Inventories437.5
417.0Prepaid expenses149.7
152.5Other current assets285.3
323.9Total current assets4,583.1
4,166.0Property, plant and equipment, net203.1
199.9Operating lease assets55.8
58.9Intangible assets, net4,203.8
4,429.5Goodwill1,805.0
1,829.3Deferred tax assets, net907.0
869.1Deferred financing costs7.1
7.6Other non-current assets94.7
99.0Total assets$ 11,859.6
$ 11,659.3LIABILITIES AND SHAREHOLDERS' EQUITY
Current liabilities:
Accounts payable$ 100.0
$ 122.1Accrued liabilities1,022.4
1,034.2Current portion of long-term debt1,030.5
1,029.9Income taxes payable93.7
56.3Total current liabilities2,246.6
2,242.5Long-term debt, less current portion4,324.2
4,328.4Operating lease liabilities, less current portion47.3
50.9Deferred tax liabilities, net547.7
594.5Other non-current liabilities161.5
124.4Total shareholders' equity4,532.3
4,318.6Total liabilities and shareholders' equity$ 11,859.6
$ 11,659.3 JAZZ PHARMACEUTICALS PLCSUMMARY OF CASH FLOWS (In millions)(Unaudited)
Three Months EndedMarch 31,
2026
2025Net cash provided by operating activities$ 408.2
$ 429.8Net cash provided by (used in) investing activities123.1
(169.0)Net cash used in financing activities(78.5)
(813.5)Effect of exchange rates on cash and cash equivalents(0.4)
1.7Net increase (decrease) in cash and cash equivalents$ 452.4
$ (551.0) JAZZ PHARMACEUTICALS PLCRECONCILIATIONS OF GAAP REPORTED TO NON-GAAP ADJUSTED INFORMATION(In millions, except per share amounts)(Unaudited)
Three Months EndedMarch 31,
2026
2025
Net
Income
Diluted
EPS
Net Income
(Loss)
Diluted
EPS/(Loss
Per Share)GAAP reported$ 293.1
$ 4.43
$ (92.5)
$ (1.52)Intangible asset amortization172.3
2.61
154.4
2.47Share-based compensation expense74.5
1.13
67.7
1.08Acquisition accounting inventory fair value step-up37.5
0.57
29.9
0.48Gain on sale of PRV(122.8)
(1.86)
—
—Income tax effect of above adjustments(35.1)
(0.54)
(54.3)
(0.87)Effect of potentially dilutive ordinary shares on non-GAAP adjusted EPS—
—
—
0.04Non-GAAP adjusted$ 419.5
$ 6.34
$ 105.2
$ 1.68Weighted-average ordinary shares used in diluted per share calculations - GAAP66.1
61.0
Dilutive effect of employee equity incentive and purchase plans—
1.6
Weighted-average ordinary shares used in diluted per share calculations - non-GAAP66.1
62.6
JAZZ PHARMACEUTICALS PLCRECONCILIATIONS OF GAAP REPORTED TO NON-GAAP ADJUSTED INFORMATION - CERTAIN LINE ITEMS(In millions, except percentages)(Unaudited)
Three months ended March 31, 2026
Cost of
product
sales
Gross
margin
SG&A
R&D
Intangible
asset
amortization
Gain on
sale
of PRV
Interest
expense,
net
Income tax
expense
Effective
tax rateGAAP Reported$ 134.1
87.5 %
$ 352.7
$ 196.0
$ 172.3
$ (122.8)
$ 39.9
$ 6.1
2.0 %Non-GAAP Adjustments:
Intangible asset amortization—
—
—
—
(172.3)
—
—
—
—Share-based compensation expense(6.6)
0.6
(44.2)
(23.7)
—
—
—
—
—Acquisition accounting inventory fair value step-up(37.5)
3.5
—
—
—
—
—
—
—Gain on sale of PRV—
—
—
—
—
122.8
—
—
—Income tax effect of above adjustments—
—
—
—
—
—
—
35.1
6.9Total of non-GAAP adjustments(44.1)
4.1
(44.2)
(23.7)
(172.3)
122.8
—
35.1
6.9Non-GAAP Adjusted$ 90.0
91.6 %
$ 308.5
$ 172.3
$ —
$ —
$ 39.9
$ 41.2
8.9 %
Three months ended March 31, 2025
Cost of
product
sales
Gross
margin
SG&A
R&D
Intangible
asset
amortization
Interest
expense,
net
Income tax
expense
(benefit)
Effective
tax rateGAAP Reported$ 104.6
88.3 %
$ 514.0
$ 180.7
$ 154.4
$ 53.7
$ (17.8)
16.2 %Non-GAAP Adjustments:
Intangible asset amortization—
—
—
—
(154.4)
—
—
—Share-based compensation expense(5.0)
0.6
(41.7)
(21.0)
—
—
—
—Acquisition accounting inventory fair value step-up(29.9)
3.3
—
—
—
—
—
—Income tax effect of above adjustments—
—
—
—
—
—
54.3
9.5Total of non-GAAP adjustments(34.9)
3.9
(41.7)
(21.0)
(154.4)
—
54.3
9.5Non-GAAP Adjusted$ 69.7
92.2 %
$ 472.3
$ 159.7
$ —
$ 53.7
$ 36.5
25.7 % JAZZ PHARMACEUTICALS PLCRECONCILIATION OF 2026 GAAP TO NON-GAAP GUIDANCE MEASURES
Projected Range(In millions, except percentages)Low
HighGAAP gross margin on total revenues89 %
90 %Acquisition accounting inventory fair value step-up1 %
1 %Non-GAAP gross margin on total revenues90 %
91 %
GAAP SG&A expenses$ 1,424
$ 1,497Share-based compensation expense(164)
(177)Non-GAAP SG&A expenses$ 1,260
$ 1,320
GAAP R&D expenses$ 811
$ 867Share-based compensation expense(86)
(92)Non-GAAP R&D expenses$ 725
$ 775
GAAP effective tax rate 0 %
10 %Income tax effect of GAAP to non-GAAP reconciling items11.5 %
3.5 %Non-GAAP effective tax rate11.5 %
13.5 %Contacts:Investors:
InvestorInfo@jazzpharma.com
Ireland +353 1 634 3211
U.S. +1 650 496 2717Media:
CorporateAffairsMediaInfo@jazzpharma.com
Ireland +353 1 637 2141
U.S. +1 215 867 4948 View original content to download multimedia:https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-announces-first-quarter-2026-financial-results-302763061.htmlSOURCE Jazz Pharmaceuticals plc Original: Jazz Pharmaceuticals Announces First Quarter 2026 Financial Results
US Market News
2月前
Jazz Pharmaceuticals Announces FDA Acceptance and Priority Review of Supplemental Biologics License Application for Ziihera® (zanidatamab-hrii) Combinations in First-Line HER2+ Locally Advanced or Metastatic GEAApril 27, 2026 7:45 AM
PR Newswire (US)
Prescription Drug User Fee Act (PDUFA) date set for August 25, 2026For U.S. media and investors onlyDUBLIN, April 27, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the U.S. Food and Drug Administration (FDA) accepted for filing with Priority Review the supplemental Biologics License Application (sBLA) for Ziihera® (zanidatamab-hrii) containing combinations for the first-line treatment of adult patients with HER2-positive (HER2+) unresectable locally advanced or metastatic gastric, gastroesophageal junction (GEJ), or gastroesophageal adenocarcinoma (GEA). The FDA has set a PDUFA target action date of August 25, 2026.The sBLA is supported by data from the pivotal HERIZON-GEA-01 trial to investigate the efficacy and safety of zanidatamab in combination with standard-of-care chemotherapy with or without the PD-1 inhibitor Tevimbra® (tislelizumab) in patients with advanced or metastatic GEA, including gastric, GEJ and esophageal adenocarcinomas. The submission is under review via the Real-Time Oncology Review (RTOR) program, an initiative of FDA's Oncology Center of Excellence designed to provide a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible."The HERIZON-GEA-01 trial results are practice changing, supporting the potential of zanidatamab as the HER2-targeted agent-of-choice in HER2+ first-line locally advanced or metastatic GEA. Importantly, the results demonstrated adding tislelizumab to zanidatamab plus chemotherapy further enhanced clinical benefit and marked the first immuno-oncology combination to show efficacy across both PD-L1–positive and PD-L1–negative tumors in this clinical setting," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of R&D, and chief medical officer of Jazz Pharmaceuticals. "We look forward to continuing to work closely with FDA to obtain approval and quickly bring zanidatamab to market for GEA patients in need of new options."The FDA granted Breakthrough Therapy designation to zanidatamab in combination with fluoropyrimidine- and platinum-containing chemotherapy, with or without tislelizumab, for the first-line treatment of patients with HER2+ unresectable locally advanced or metastatic gastric, GEJ, or esophageal adenocarcinoma. Breakthrough Therapy designation is intended to expedite the development and review of therapies that, based on preliminary clinical evidence, may offer substantial improvement over available therapies on one or more clinically significant endpoints, reflecting both the seriousness of the disease and the unmet medical need in this setting.About the Phase 3 HERIZON-GEA-01 Trial
HERIZON-GEA-01 (NCT05152147) is a global, randomized, open-label Phase 3 trial, conducted jointly with BeOne Medicines, to evaluate and compare the efficacy and safety of zanidatamab plus chemotherapy, with or without tislelizumab, to trastuzumab plus chemotherapy as first-line treatment for adult patients with advanced/metastatic HER2+ GEA. The trial randomized 914 patients from approximately 300 trial sites in more than 30 countries. Appropriate patients for this trial had unresectable locally advanced, recurrent or metastatic HER2+ GEA (adenocarcinomas of the stomach or esophagus, including the gastroesophageal junction), defined as 3+ HER2 expression by IHC or 2+ HER2 expression by IHC with ISH positivity per central assessment. Patients were randomized to the three trial arms: zanidatamab in combination with chemotherapy and tislelizumab; zanidatamab in combination with chemotherapy; and trastuzumab plus chemotherapy. The trial is evaluating dual primary endpoints, PFS per blinded independent central review (BICR) and OS. Results from the trial were presented in January 2026 at the 2026 ASCO Gastrointestinal Cancers Symposium.About Gastroesophageal Adenocarcinoma
GEA, including cancers of the stomach, gastroesophageal junction, and esophagus, is the fifth most common cancer worldwide, and approximately 20% of patients have HER2+ disease.1,2,3 HER2+ GEA has high morbidity and mortality, and patients are urgently in need of new treatment options. The overall prognosis for patients with GEA remains poor, with a global five-year survival rate of less than 30% for gastric cancer and about 19% for GEA.4About Ziihera® (zanidatamab-hrii)
Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab-hrii induces complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC), and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.5 In the United States, Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.1 The FDA granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).1 Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz Pharmaceuticals and BeOne under license agreements from Zymeworks, which first developed the molecule. An sBLA for zanidatamab is being reviewed by the FDA under RTOR in first-line HER2+ locally advanced or metastatic GEA. The FDA granted two Breakthrough Therapy designations for zanidatamab's development: one as a single agent for previously treated HER2 gene-amplified BTC, and one in combination with fluoropyrimidine- and platinum-containing chemotherapy, with or without tislelizumab for first-line HER2+ unresectable locally advanced or metastatic gastric, GEJ or esophageal adenocarcinoma. The FDA also granted two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for first-line GEA. Additionally, zanidatamab has received Orphan Drug designations from the FDA for the treatment of BTC, gastric (including GEJ) cancer, and esophageal cancer, as well as Orphan Drug designations from the European Medicines Agency for the treatment of BTC, gastric/GEJ cancer and oesophageal cancer. Important Safety Information for ZIIHERA WARNING: EMBRYO-FETAL TOXICITY
Exposure to ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patients
of the risk and need for effective contraception. WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity
ZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death. Verify the pregnancy status of females of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA. Left Ventricular Dysfunction
ZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to 2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA. The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%). USE IN SPECIFIC POPULATIONS Pediatric Use
Safety and efficacy of ZIIHERA have not been established in pediatric patients. Geriatric Use
Of the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older. No overall differences in safety or efficacy were observed between these patients and younger adult patients. The full U.S. Prescribing Information for ZIIHERA, including BOXED Warning, is available at: https://pp.jazzpharma.com/pi/ziihera.en.USPI.pdf ® TEVIMBRA (tislelizumab) is a registered trademark of BeOne Medicines.About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with rare disease — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies addressing epilepsies, cancers and sleep disorders. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information. Cautionary Note Concerning Forward-Looking Statements
This press release contains forward-looking statements, including, but not limited to, statements related to the potential therapeutic benefits of Ziihera (zanidatamab-hrii) and of combination therapies with zanidatamab, zanidatamab's potential as a new standard of care in HER2+ first-line GEA and other HER2-expressing cancers, expected timing of and ability to obtain FDA approval under the RTOR program for zanidatamab in HER2+ first-line GEA and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the successful completion of regulatory activities and uncertain regulatory approval, risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals' Securities and Exchange Commission filings and reports, including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2025, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts:
Media:
CorporateAffairsMediaInfo@jazzpharma.com
Ireland +353 1 637 2141
U.S. +1 215 867 4948Investor:
InvestorInfo@jazzpharma.com
Ireland +353 1 634 3211
U.S. +1 650 496 27171Abrahao-Machado I.F., et al. HER2 testing in gastric cancer: An update WorldJGastroenterol. 2016;22(19):4619-4625.
2Van Custem E., et al. HER2 screening data from ToGA: targeting HER2 in gastric and gastroesophageal junction cancer. Gastric Cancer. 2015;18(3):476-484.
3Stroes, C.I., et al. A systematic review of HER2 blockade for the curative treatment of gastroesophageal adenocarcinoma: Successes achieved and opportunities ahead. CancerTreatRev. 2021;99:102249.
4Battaglin F, et al. Molecular biomarkers in gastro-esophageal cancer: recent developments, current trends and future directions. Cancer Cell International. 2018;18(99).
5ZIIHERA (zanidatamab-hrii) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.
View original content to download multimedia:https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-announces-fda-acceptance-and-priority-review-of-supplemental-biologics-license-application-for-ziihera-zanidatamab-hrii-combinations-in-first-line-her2-locally-advanced-or-metastatic-gea-302753741.htmlSOURCE Jazz Pharmaceuticals plc
Original: Jazz Pharmaceuticals Announces FDA Acceptance and Priority Review of Supplemental Biologics License Application for Ziihera® (zanidatamab-hrii) Combinations in First-Line HER2+ Locally Advanced or Metastatic GEA
US Market News
3月前
Jazz Pharmaceuticals to Present Data at ASCO 2026 Highlighting Advancements for Ziihera® (zanidatamab-hrii) in Gastroesophageal Adenocarcinoma and Zepzelca® (lurbinectedin)April 21, 2026 4:05 PM
PR Newswire (US)
Rapid oral presentation of PD-L1 subgroup data from HERIZON-GEA-01 evaluating zanidatamab combinations, and additional analyses of tolerability, biomarker response and real-world treatment patterns in HER2+ GEAFor U.S. media and investors onlyDUBLIN, April 21, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Company and its partners will present three rapid oral and seven poster presentations at the American Society of Clinical Oncology (ASCO) Annual Meeting, taking place from May 29-June 2, 2026, in Chicago. The data reflect Jazz's continued momentum in oncology and the Company's focus on advancing treatment approaches in difficult-to-treat cancers through late-stage clinical research, real-world evidence and ongoing pipeline innovation.Key ASCO 2026 presentations include:Data from the Phase 3 HERIZON-GEA-01 trial evaluating Ziihera® (zanidatamab-hrii) in combination with chemotherapy with or without the PD-1 inhibitor Tevimbra® (tislelizumab) in previously untreated HER2-positive (HER2+) gastroesophageal adenocarcinoma (GEA), including a rapid oral presentation of analyses of progression-free survival (PFS) and overall survival (OS) across PD-L1 subgroups, as well as analyses of the characterization and management of gastrointestinal adverse events.Analyses from the Phase 3 IMforte trial evaluating Zepzelca® (lurbinectedin) plus atezolizumab (Tecentriq®) as first-line maintenance treatment in extensive-stage small cell lung cancer (ES-SCLC), including quality-adjusted time without symptoms or toxicity, as well as a rapid oral presentation on outcomes across SCLC molecular subtypes."Building on the strength of the HERIZON-GEA-01 trial results, additional analyses being presented at ASCO will provide further details on the impact of zanidatamab in HER2+ GEA, including across PD-L1 subgroups. These data will continue to inform treatment decision-making and enable the successful treatment integration of zanidatamab in clinical practice," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "Together with additional analyses of Zepzelca from the Phase 3 IMforte study in first-line maintenance extensive-stage small cell lung cancer and progress in our pipeline, these data reflect our growing and increasingly differentiated oncology portfolio, as well as our commitment to advancing innovative approaches for patients facing some of the most difficult-to-treat cancers."The ASCO abstracts are available at: https://meetings.asco.org/meetings/2026-asco-annual-meeting/335/program-guide/scheduled-sessionsThe full list of Jazz- and partner-supported presentations at the 2026 ASCO Annual Meeting are:Zanidatamab Presentations:Presentation TitleAuthorsPresentation DetailsMolecular circulating
tumor DNA (ctDNA)
profiling from patients
(pts) treated with
zanidatamab +
chemotherapy (CT) in
first-line (1L) HER2-
positive (HER2+)
advanced or
metastatic
gastroesophageal
adenocarcinoma (mGEA) Elimova E, Ku GY, Lee KW, Rha SY,
Wienke S, Yalamanchili G, Garfin PM,
Loro E, Shpektor D, Ajani JAType: PosterSession: Poster Session –
Gastrointestinal Cancer-
Gastroesophageal, Pancreatic,
and HepatobiliaryDate/Time: May 30, 2026,
9 a.m.-Noon CDTAbstract number: 4050Real-world treatment
patterns and overall
survival (OS) in
patients (pts) with
HER2-positive
(HER2+) advanced or
metastatic
gastroesophageal
adenocarcinomas
(mGEA) in the USDayyani F, Fan X, Zape J, Murphy R,
Betts KA, Wang Y, Wang S, Chao A, Su
W, Fuller DS, Sabater J, Gibson MK,
Enzinger PC Type: PosterSession: Poster Session –
Gastrointestinal Cancer-
Gastroesophageal, Pancreatic,
and HepatobiliaryDate/Time: May 30, 2026, 9
a.m.-Noon CDTAbstract number: 4053Characterization and
management of
gastrointestinal (GI)
adverse events (AEs)
with zanidatamab +
chemotherapy (CT) ±
tislelizumab in first-line
(1L) HER2-positive
(HER2+) locally
advanced or
metastatic
gastroesophageal
adenocarcinoma
(mGEA): Analysis
from HERIZON-GEA-
01 Elimova E, Rha SY, Shitara K, Liu T,
Tabernero J, Lee KW, Schenker M,
Tebbutt NC, Ajani JA, Salimin N, Ku GY,
Kim JG, Diaz IA, Zhang J, Pietrantonio F,
Bai LY, Le Sourd SL, Chen Y, Grim JE,
Shen LType: PosterSession: Poster Session –
Gastrointestinal Cancer-
Gastroesophageal, Pancreatic,
and HepatobiliaryDate/Time: May 30, 2026, 9
a.m.-Noon CDTAbstract number: 4042Combining
zanidatamab,
FOLFOX, and
pembrolizumab as
first-line therapy for
HER2/PD-L1-positive
gastroesophageal
adenocarcinoma –
The phase ll IKF-
090/AIO ZANGEA trial
with translational
analysisTintelnot J, Goekkurt E, Al-Batran SE,
Arnold D, Dechow TN, Ettrich TJ, Goetze
TO, Heinrich K, Kurreck A, Lorenzen S,
Moehler MH, Rempel V, Schlenska-Lange
A, Stein AType: PosterSession: Poster Session –
Gastrointestinal Cancer-
Gastroesophageal, Pancreatic,
and HepatobiliaryDate/Time: May 30, 2026, 9
a.m.-Noon CDTAbstract number: TPS4244Zanidatamab +
chemotherapy (CT) ±
tislelizumab for first-
line (1L) HER2-
positive (HER2+)
locally advanced or
metastatic
gastroesophageal
adenocarcinoma
(mGEA): PD-L1
subgroup analysis
from HERIZON-GEA
-01Rha SY, Shitara K, Shen L, Tabernero J,
Liu T, Lee KW, Schenker M, Tebbutt NC,
Ajani JA, Salimin N, Ku GY, Kim JG, Diaz
IA, Zhang J, Pietrantonio F, Bai LY, Sourd
SL, Chen Y, Grim JE, Elimova EType: Rapid OralSession: Rapid Oral Abstract
Session – Gastrointestinal
Cancer-Gastroesophageal,
Pancreatic, and HepatobiliaryDate/Time: June 1, 2026, 1:15-2:45 p.m. CDTAbstract number: 4010Lurbinectedin Presentations:Presentation TitleAuthorsPresentation DetailsReal-world (RW)
effectiveness and
safety of lurbinectedin
(lurbi) for previously
treated extensive-
stage small cell lung
cancer (ES-SCLC):
Final primary and
subgroup analysis
results of Jazz
EMERGE 402Badin FB, Lammers PE, Liu G,
Shunyakov L, Kassam SN, Patel MP, Ji Y,
Labbé C, Rabara V, Hashmi MH, Dakhil
SR, Weiss M, Gowan AC, Bouchard N,
Rengarajan B, Fuller DS, Naveh N,
Halmos BType: PosterSession: Poster Session –
Lung Cancer-Non-Small Cell
Local-Regional/Small Cell/Other
Thoracic CancersDate/Time: May 31, 2026, 9
a.m.-Noon CDTAbstract number: 8079Comparison of real-
world overall survival
between
atezolizumab- and
durvalumab-containing
first-line induction and
maintenance regimens
in extensive stage
small cell lung cancerGanti AK, Snider J, Yan J, Rinaldi C,
Nguyen A, Rengarajan B, Profant DA,
Fuller DS, Hu E, Le TK, Naveh N, Fan XType: PosterSession: Poster Session –
Lung Cancer-Non-Small Cell
Local-Regional/Small Cell/Other
Thoracic CancersDate/Time: May 31, 2026, 9
a.m.-Noon CDTAbstract number: 8093IMforte: Quality
-adjusted time without
symptoms or toxicity
(Q-TWiST) analysis of
first-line maintenance
(1Lm) treatment (Tx)
with lurbinectedin
(lurbi) + atezolizumab
(atezo) vs atezo in
extensive-stage small
cell lung cancer (ES-
SCLC)Borghaei H, Paz-Ares LG, Reck M, Herbst
RS, Peters S, Bhatt K, Wang X, Gable J,
Connor-Ahmad S, Mamolo C, Lin YC, Liu
SVType: PosterSession: Poster Session –
Lung Cancer-Non-Small Cell
Local-Regional/Small Cell/Other
Thoracic CancersDate/Time: May 31, 2026, 9
a.m.-Noon CDTAbstract number: 8086Transcriptomic
analyses of molecular
subsets and
correlations with
clinical outcomes from
the Phase 3 IMforte
study of lurbinectedin
(lurbi) + atezolizumab
(atezo) maintenance
treatment (Tx) in
extensive-stage small-
cell lung cancer (ES-
SCLC)Paz-Ares L, Borghaei H, Reck M, Peters
S, Herbst RS, Kazarnowicz A, Szczesna
A, Cubukcu E, Kilickap S, Ahn JS,
Califano R, Wei YF, Srivastava MK, Nabet
BY, Graupner V, Lin YC, Cai G, Brock G,
Bhatt K, Liu SVType: Rapid OralSession: Rapid Oral Abstract
Session – Lung Cancer-Non-
Small Cell Local-Regional/Small
Cell/Other Thoracic CancersDate/Time: May 31, 2026, 4:30-
6 p.m. CDTAbstract number: 8014Safety and
pharmacokinetics (PK)
of lurbinectedin (lurbi)
in pediatric patients
(pts) with
relapsed/refractory
(R/R) solid tumors and
preliminary antitumor
activity in pediatric and
young adult pts with
R/R Ewing sarcoma
(EwS): Results from a
phase 1 studyGlade Bender JL, Pressey JG, Wagner
LM, Kim AR, Shah AT, Federico SM,
Morgenstern DA, Hoogstra DJ, Crane J,
Bhatt K, Prakash R, Faderl S, Parikh P,
Daniels M, Shi S, Wang X, Cai G, Miao X,
Ma J, Laetsch TWType: Rapid OralSession: Rapid Oral Abstract
Session – Sarcoma Date/Time: May 31, 2026, 4:30-
6 p.m. CDTAbstract number: 11518About Ziihera® (zanidatamab-hrii)
Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab-hrii induces complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC), and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.[1] In the United States, Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.1 The FDA granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).1 Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by?Jazz?and BeOne under license agreements from Zymeworks, which first developed the molecule.?? A supplemental biologics license application for zanidatamab was submitted to the FDA under Real-Time Oncology Review in first-line HER2+ locally advanced or metastatic GEA. The FDA granted two Breakthrough Therapy designations for zanidatamab's development: one as a single agent for previously treated HER2 gene-amplified BTC, and one in combination with fluoropyrimidine- and platinum-containing chemotherapy, with or without tislelizumab for first-line HER2+ unresectable locally advanced or metastatic gastric, gastroesophageal junction (GEJ) or esophageal adenocarcinoma. The FDA also granted two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for first-line GEA. Additionally, zanidatamab has received Orphan Drug designations from the FDA for the treatment of BTC, gastric (including GEJ) cancer, and esophageal cancer, as well as Orphan Drug designations from the European Medicines Agency for the treatment of BTC, gastric/gastroesophageal junction cancer and oesophageal cancer. Important Safety Information for ZIIHERA WARNING: EMBRYO-FETAL?TOXICITY
Exposure to?ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patients
of the risk and need for effective contraception. WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity
ZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death. Verify the pregnancy status of females of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA. Left Ventricular Dysfunction
ZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to 2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA. The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%). USE IN SPECIFIC POPULATIONS Pediatric Use
Safety and efficacy of ZIIHERA have not been established in pediatric patients. Geriatric Use
Of the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older. No overall differences in safety or efficacy were observed between these patients and younger adult patients. The full U.S. Prescribing Information for ZIIHERA, including BOXED Warning, is available at: https://pp.jazzpharma.com/pi/ziihera.en.USPI.pdf About Zepzelca® (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and potentially cell death.2In October 2025, the FDA approved Zepzelca in combination with atezolizumab or atezolizumab and hyaluronidase-tqjs, as maintenance treatment for adults with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab or atezolizumab and hyaluronidase-tqjs, carboplatin and etoposide.In June 2020, the FDA approved Zepzelca under accelerated approval for the treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. This indication is approved under accelerated approval based on ORR and DOR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).Important Safety Information for ZEPZELCA Myelosuppression
ZEPZELCA can cause severe and fatal myelosuppression including febrile neutropenia and sepsis, thrombocytopenia and anemia.Administer ZEPZELCA only to patients with baseline neutrophil count of at least 1,500 cells/mm3 and platelet count of at least 100,000/mm3. To reduce the risk of febrile neutropenia during treatment with ZEPZELCA in combination with atezolizumab or atezolizumab and hyaluronidase-tqjs, administer granulocyte colony-stimulating factor (G-CSF). Monitor blood counts including neutrophils, red blood cells and platelets prior to each ZEPZELCA administration. For neutrophil count less than 500 cells/mm3 or any value less than lower limit of normal, the use of G-CSF is recommended. Withhold, reduce the dose, or permanently discontinue ZEPZELCA based on severity.ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, primary prophylaxis of G-CSF was administered to 84% of patients. Based on laboratory values, decreased neutrophils occurred in 36%, including 18% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. The median time to onset of Grade 3 and 4 decreased neutrophil cells was 31 days and a median duration of 10 days. Febrile neutropenia occurred in 1.7%. Sepsis occurred in 1%. There were 7 fatal infections: pneumonia (n=3), sepsis (n=3), and febrile neutropenia (n=1).Based on laboratory values, decreased platelets occurred in 54%, including 15% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. The median time to onset of Grade 3 and 4 decreased platelet cells was 31 days and a median duration of 12 days.Based on laboratory values, decreased hemoglobin occurred in 51%, including 13% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. The median time to onset of Grade 3 and 4 decreased hemoglobin was 64 days and a median duration of 8 days.ZEPZELCA as a Single AgentIn clinical studies of 554 patients with advanced solid tumors receiving ZEPZELCA as a single agent, Grade 3 or 4 neutropenia occurred in 41% of patients, with a median time to onset of 15 days and a median duration of 7 days. Febrile neutropenia occurred in 7% of patients. Sepsis occurred in 2% of patients and was fatal in 1% (all cases occurred in patients with solid tumors other than SCLC). Grade 3 or 4 thrombocytopenia occurred in 10%, with a median time to onset of 10 days and a median duration of 7 days. Grade 3 or 4 anemia occurred in 17% of patients.Hepatotoxicity
ZEPZELCA can cause hepatotoxicity which may be severe.Monitor liver function tests prior to initiating ZEPZELCA and periodically during treatment as clinically indicated. Withhold, reduce the dose, or permanently discontinue ZEPZELCA based on severity.ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, based on laboratory values, increased alanine aminotransferase (ALT) occurred in 25%, including 3% Grade 3 or Grade 4 in patients who received ZEPZELCA in combination with atezolizumab. Increased aspartate aminotransferase (AST) occurred in 24% including 3% Grade 3 or Grade 4. The median time to onset of Grade ≥3 elevation in transaminases was 52 days (range: 6 to 337).ZEPZELCA as a Single AgentIn clinical studies of 554 patients with advanced solid tumors receiving ZEPZELCA as a single agent, Grade 3 elevations of ALT and AST were observed in 6% and 3% of patients, respectively, and Grade 4 elevations of ALT and AST were observed in 0.4% and 0.5% of patients, respectively. The median time to onset of Grade ≥3 elevation in transaminases was 8 days (range: 3 to 49), with a median duration of 7 days.Extravasation Resulting in Tissue Necrosis
Extravasation of ZEPZELCA can cause skin and soft tissue injury, including necrosis requiring debridement. Consider use of a central venous catheter to reduce the risk of extravasation, particularly in patients with limited venous access. Monitor patients for signs and symptoms of extravasation during the ZEPZELCA infusion. If extravasation occurs, immediately discontinue the infusion, remove the infusion catheter, and monitor for signs and symptoms of tissue necrosis. The time to onset of necrosis after extravasation may vary.ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, extravasation resulting in skin necrosis occurred in one patient who received ZEPZELCA in combination with atezolizumab.Administer supportive care and consult with an appropriate medical specialist as needed for signs and symptoms of extravasation. Administer subsequent infusions at a site that was not affected by extravasation. Rhabdomyolysis
Rhabdomyolysis has been reported in patients treated with ZEPZELCA.Monitor creatine phosphokinase (CPK) prior to initiating ZEPZELCA and periodically during treatment as clinically indicated. Withhold or reduce the dose based on severity. ZEPZELCA with Intravenous AtezolizumabIn the IMforte study, among 235 patients who had a creatine phosphokinase laboratory evaluation, increased creatine phosphokinase occurred in 9% who received ZEPZELCA in combination with atezolizumab.Embryo-Fetal Toxicity
ZEPZELCA can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise female patients of reproductive potential to use effective contraception during treatment with ZEPZELCA and for 7 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ZEPZELCA and for 4 months after the last dose.Lactation
There are no data on the presence of ZEPZELCA in human milk, however, because of the potential for serious adverse reactions from ZEPZELCA in breastfed children, advise women not to breastfeed during treatment with ZEPZELCA and for 2 weeks after the last dose.ADVERSE REACTIONSZEPZELCA with Intravenous AtezolizumabSerious adverse reactions occurred in 31% of patients receiving ZEPZELCA in combination with atezolizumab. Serious adverse reactions occurring in >2% were pneumonia (2.5%), respiratory tract infections (2.1%), dyspnea (2.1%), and decreased platelet count (2.1%). Fatal adverse reactions occurred in 5% of patients receiving ZEPZELCA with atezolizumab including pneumonia (3 patients), sepsis (3 patients), cardio-respiratory arrest (2 patients), myocardial infarction (2 patients), and febrile neutropenia (1 patient).The most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received ZEPZELCA with atezolizumab were decreased lymphocytes (55%), decreased platelets (54%), decreased hemoglobin (51%), decreased neutrophils (36%), nausea (36%), and fatigue/asthenia (32%).ZEPZELCA as a Single AgentSerious adverse reactions occurred in 34% of patients who received ZEPZELCA. Serious adverse reactions in ≥3% of patients included pneumonia, febrile neutropenia, neutropenia, respiratory tract infection, anemia, dyspnea, and thrombocytopenia.The most common adverse reactions, including laboratory abnormalities, (≥20%) are leukopenia (79%), lymphopenia (79%), fatigue (77%), anemia (74%), neutropenia (71%), increased creatinine (69%), increased alanine aminotransferase (66%), increased glucose (52%), thrombocytopenia (37%), nausea (37%), decreased appetite (33%), musculoskeletal pain (33%), decreased albumin (32%), constipation (31%), dyspnea (31%), decreased sodium (31%), increased aspartate aminotransferase (26%), vomiting (22%), decreased magnesium (22%), cough (20%), and diarrhea (20%).DRUG INTERACTIONS
Effect of CYP3A Inhibitors and Inducers
Avoid coadministration with a strong or a moderate CYP3A inhibitor (including grapefruit and Seville oranges) as this increases lurbinectedin systemic exposure which may increase the incidence and severity of adverse reactions to ZEPZELCA. If coadministration cannot be avoided, reduce the ZEPZELCA dose as appropriate.Avoid coadministration with a strong CYP3A inducer as it may decrease systemic exposure to lurbinectedin, which may decrease the efficacy of ZEPZELCA.GERIATRIC USEZEPZELCA with Intravenous AtezolizumabOf the 242 patients with ES-SCLC treated with ZEPZELCA and atezolizumab in IMforte, 124 (51%) patients were 65 years of age and older, while 29 (12%) patients were 75 years of age and older. No overall differences in effectiveness were observed between older and younger patients. There was no overall difference in the incidence of serious adverse reactions in patients ≥65 years of age and patients
US Market News
3月前
Jazz Pharmaceuticals to Present Research on Epidiolex® (cannabidiol) and Xywav® (calcium, magnesium, potassium, and sodium oxybates) Oral Solution at the 2026 American Academy of Neurology Annual MeetingApril 9, 2026 7:45 AM
PR Newswire (US)
Six abstracts, including one oral presentation, underscore Jazz's ongoing commitment to generating real-
world evidence addressing rare neurological disorders, including rare and severe forms of epilepsy and
sleep disordersFor U.S. media and investors onlyDUBLIN, April 9, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Company will present six abstracts, including one oral presentation, at the 2026 American Academy of Neurology (AAN) Annual Meeting, taking place April 18-22, 2026, in Chicago.The research demonstrates the breadth of Jazz's neuroscience portfolio spanning epilepsy and sleep disorders, with new clinical data evaluating Epidiolex® (cannabidiol) in patients with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC), or other refractory epilepsies; and Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution in patients with narcolepsy."Jazz is proud to present research at another AAN Annual Meeting, especially as we continue to sharpen our focus on the significant unmet needs faced by people with rare neurological disorders, including epilepsy and sleep disorders," said Jessa Alexander, Ph.D., neuroscience therapeutic area head, global medical and scientific affairs of Jazz Pharmaceuticals. "Through our ongoing Epidiolex and Xywav research, we are gaining a deeper understanding of these complex conditions, and the real treatment needs patients experience, with data that enriches our view of the patient journey and focuses our R&D efforts on closing persistent gaps in care across rare neurology."Highlights at the 2026 AAN Annual Meeting include:An oral presentation showcasing results from the prespecified 6-month intermediate analysis of the ongoing EpiCom Phase 3b/4 study demonstrated reductions in the severity of caregiver- and clinician-reported behavioral problems at 26 weeks after Epidiolex treatment initiation in patients with TSC-associated seizures. Improvements were seen across the TSC-Associated Neuropsychiatric Disorders Self-Report Quantified Checklist (TAND-SQ), Aberrant Behavior Checklist (ABC) subscale scores and Caregiver- and Clinician-Global Impression of Severity (CGI-S) scales.A poster presentation detailing sociodemographic and clinical factors associated with persistence on Epidiolex among patients with LGS, DS, TSC, or other refractory epilepsies, based on claims analysis. This analysis of over 7,800 patients demonstrates how care variability in a non-standardized and fragmented care environment like epilepsy sheds light on the key factors that may influence whether patients remain on treatment and may help clinicians support treatment continuity.A poster presentation featuring an analysis of adults with narcolepsy from the open-label, single-arm DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) study evaluating changes in cognitive complaints, daily functioning, work productivity and overall disease burden in patients treated with Xywav dosages >9 grams per night. Improvements in these outcomes were observed in participants who optimized their Xywav dosage to >9 grams per night (up to 12 grams total) when compared to a 9 gram per night dosage. The current recommended dosage of Xywav for adults with narcolepsy is 6-9 grams per night.The 2026 AAN Annual Meeting abstracts are available online at: https://index.mirasmart.com/AAN2026/.The full list of Jazz's presentations are:Presentation TitleAuthorsPresentation DetailsEpidiolex PresentationsTuberous Sclerosis Complex
(TSC)–Associated Neuropsychiatric
Disorders (TAND) Outcomes
Following Adjunctive Cannabidiol
(CBD) Treatment: 6-Month
Intermediate Analysis of the
EpiCom TrialAgnies van Eeghen, Sarah
M.L. Wilson, Elizabeth A.
Thiele, Stevie Roszkowski,
Maria Dunaway-Bryant,
Kasia Wajer, Teresa Greco,
Joanne Stevens, Lisa
Moore-Ramdin, Petrus J.
de Vries Type: Oral PresentationSession: S5: Clinical EpilepsyDate/Time: Sunday, April 19 at
1:48 p.m. CTPresentation Number: 005An Assessment of Acute
Pharmacodynamic Drug-Drug
Interactions (PD-DDIs) Between
Cannabidiol (CBD) and
Cenobamate (CNB) in a Mouse
Model of Generalized Tonic
Seizures Rohini R. Rana, William H.
Hind, Pabitra H. Patra,
Saga Johansson, Karthik
Rajasekaran, H. Steve
White, David J. Virley Type: PosterSession: P3 Epilepsy/Clinical
Neurophysiology (EEG): Anti-
seizure Medications: Mechanisms
and PharmacologyDate/Time: Sunday, April 19,
5-6 p.m. CT Abstract Number: 001Demographics and Clinical Factors
Associated With Persistence on
Cannabidiol (CBD) in US Patients
With Lennox-Gastaut Syndrome
(LGS), Dravet Syndrome (DS),
Tuberous Sclerosis Complex
(TSC), or Other Refractory
Epilepsies Leah Burn, Vicki Osborne,
Arthur Sillah, Timothy
Saurer, Michael Faithe,
Maggie McCarter, Sanket
Shah, Timothy Barnes,
Arunima Sachdev, Anuj
Gupta, Mohankumar
Kurukumbi Type: PosterSession: P10 Epilepsy/Clinical
Neurophysiology (EEG): Epilepsy
Syndromes and GeneticsDate/Time: Wednesday, April 22,
8-9 a.m. CTAbstract Number: 004Cannabidiol Efficacy in Patients
With Lennox-Gastaut Syndrome
With Developmental and Epileptic
Encephalopathy-Associated
Genetic Variants: A Subgroup
Analysis Elizabeth A. Thiele, Michael
Boffa, Daniel Checketts,
Farhad Sahebkar
Type: PosterSession: P10 Epilepsy/Clinical
Neurophysiology (EEG): Epilepsy
Syndromes and GeneticsDate/Time: Wednesday, April 22,
8-9 a.m. CTAbstract Number: 008Phase 1, Open-Label, Fixed-
Sequence, Bidirectional,
Pharmacokinetic (PK) Drug-Drug
Interaction (DDI) Study Between
Cannabidiol (CBD) and
Cenobamate (CNB) in Healthy
Adult Participants
Arjun Vijan, Hongwei Xue,
Patricia Chandler, Sujith
Madhavan, Cuiping Chen Type: PosterSession: P11 Epilepsy/Clinical
Neurophysiology (EEG): Anti-
seizure Medications: Clinical
TrialsDate/Time: Wednesday, April 22,
11:45 a.m. - 12:45 p.m. CTAbstract Number: 002Xywav PresentationChanges in Self-Reported Cognitive
Complaints, Functional
Impairments, Work Productivity,
and Symptom Severity in
Participants With Narcolepsy After
Treatment With Greater Than 9
Gram Dosages of Low-Sodium
Oxybate in the DUET StudyLogan Schneider, Chad
Ruoff, David Plante, Jerald
H. Simmons, Deborah A.
Nichols, Teresa Steininger,
Marisa Whalen, Jing Dai,
Alyssa Cairns, Richard K.
BoganType: PosterSession: P3: Sleep:
PathophysiologyDate/Time: Sunday, April 19,
5-6 p.m. CTAbstract Number: 003About Epidiolex®/Epidyolex® (cannabidiol)
Epidiolex/Epidyolex is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient in Epidiolex, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which Epidiolex exerts its anticonvulsant effect in humans are unknown. Epidiolex has been approved for use by healthcare regulatory authorities in the U.S., Europe, Great Britain, Canada, Australia, Switzerland, Taiwan, Israel, and New Zealand, among other countries. In the U.S., Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC) in patients one year of age and older. Epidiolex has received approval in the European Union under the tradename Epidyolex for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and DS in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older. Epidiolex has received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of seizures associated with LGS, DS, and TSC. Similarly, Epidyolex received ODD from the European Medicines Agency (EMA) for the same indications. Epidiolex is also being studied in additional forms of epilepsy, including the EpiFOS exploratory study (NCT07233239) in focal-onset seizures.Important Safety Information & Indications CONTRAINDICATION: HYPERSENSITIVITY EPIDIOLEX (cannabidiol) oral solution is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product.WARNINGS & PRECAUTIONS Hepatic Injury:
EPIDIOLEX can cause dose-related transaminase elevations. Concomitant use of valproate and elevated transaminase levels at baseline increase this risk. Obtain transaminase and bilirubin levels prior to starting treatment, at 1, 3, and 6 months after initiation of treatment, and periodically thereafter, or as clinically indicated. Resolution of transaminase elevations occurred with discontinuation of EPIDIOLEX, reduction of EPIDIOLEX and/or concomitant valproate, or without dose reduction. For patients with elevated transaminase levels, consider dose reduction or discontinuation of EPIDIOLEX or concomitant medications known to affect the liver (e.g., valproate or clobazam). Dose adjustment and slower dose titration is recommended in patients with moderate or severe hepatic impairment. Consider not initiating EPIDIOLEX in patients with evidence of significant liver injury. There have been postmarketing reports of cholestatic or mixed patterns of liver injury. Elevated ammonia levels were reported in some patients with transaminase elevations; most taking concomitant valproate, clobazam, or both. Consider discontinuation or dose adjustment of valproate or clobazam if ammonia is elevated.Somnolence and Sedation:
EPIDIOLEX can cause somnolence and sedation that generally occurs early in treatment and may diminish over time; these effects occur more commonly in patients using clobazam and may be potentiated by other CNS depressants.Suicidal Behavior and Ideation:
Antiepileptic drugs (AEDs), including EPIDIOLEX, increase the risk of suicidal thoughts or behavior. Inform patients, caregivers, and families of the risk and advise them to monitor and report any signs of depression, suicidal thoughts or behavior, or unusual changes in mood or behavior. If these symptoms occur, consider if they are related to the AED or the underlying illness.Withdrawal of Antiepileptic Drugs:
As with most AEDs, EPIDIOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus.ADVERSE REACTIONS:
The most common adverse reactions in patients receiving EPIDIOLEX (≥10% and greater than placebo) include transaminase elevations; somnolence; decreased appetite; diarrhea; pyrexia; vomiting; fatigue, malaise, and asthenia; rash; insomnia, sleep disorder and poor-quality sleep; and infections. Hematologic abnormalities were also observed.PREGNANCY:
EPIDIOLEX should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Encourage women who are taking EPIDIOLEX during pregnancy to enroll in the EPIDIOLEX Pregnancy Surveillance Program and the North American Antiepileptic Drug (NAAED) Pregnancy Registry.DRUG INTERACTIONS:
Strong inducers of CYP3A4 and CYP2C19 may affect EPIDIOLEX exposure. EPIDIOLEX may affect exposure to CYP2C19 substrates (e.g., clobazam, diazepam, stiripentol), orally administered P-gp substrates, or other substrates (see full Prescribing Information). Consider dose reduction of orally administered everolimus, with appropriate therapeutic drug monitoring, when everolimus is combined with EPIDIOLEX. A lower starting dose of everolimus is recommended when added to EPIDIOLEX therapy. Concomitant use of EPIDIOLEX and valproate increases the incidence of liver enzyme elevations. Pneumonia was observed more frequently with concomitant use of EPIDIOLEX and clobazam. Dosage adjustment of EPIDIOLEX or other concomitant medications may be necessary.INDICATIONS:
EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older.Please read the EPIDIOLEX full Prescribing Information for additional important information here. About Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution
Xywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy. The Office of Orphan Product Development (OOPD) at the FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy by means of greater cardiovascular safety compared to Xyrem® (sodium oxybate) oral solution. The decision of the OOPD is based on the FDA findings that Xywav provides a greatly reduced chronic sodium burden compared to Xyrem. Xywav has 131 mg of sodium at the maximum recommended nightly dose whereas other high sodium oxybates have 1640 mg at the equivalent dose. Xywav is comprised of a unique composition of cations resulting in 92% less sodium, or a reduction of approximately 1,000 to 1,500 mg/night at the recommended dose range of 6 g to 9 g/night. Xywav is the only oxybate therapy that does not carry a warning in the label related to use in patients sensitive to high sodium intake.Xywav is also the first and only U.S. FDA-approved treatment option for idiopathic hypersomnia in adults. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of idiopathic hypersomnia in adults. Xywav is the only FDA-approved treatment studied across the multiple symptoms of idiopathic hypersomnia, such as EDS, sleep inertia (severe grogginess or confusion when waking up), long sleep duration and cognitive impairment. Xywav can be administered as a twice- or once-nightly regimen for the treatment of idiopathic hypersomnia in adults.The exact mechanism of action of Xywav in the treatment of adults with idiopathic hypersomnia and of cataplexy and EDS in narcolepsy is unknown. It is hypothesized that the therapeutic effects of Xywav are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as thalamocortical neurons.1 The U.S. Drug Enforcement Agency (DEA) has designated Xywav as a Schedule III medicine. The DEA defines Schedule III drugs, substances, or chemicals as drugs with a moderate to low potential for physical and psychological dependence.1,2 Because of the risks of central nervous system (CNS) depression and abuse and misuse, Xywav is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS.Important Safety Information for XywavWARNING: CENTRAL NERVOUS SYSTEM DEPRESSION and ABUSE AND MISUSE.Central Nervous System Depression
XYWAV is a CNS depressant. Clinically significant respiratory depression and
obtundation may occur in patients treated with XYWAV at recommended doses. Many
patients who received XYWAV during clinical trials in narcolepsy and idiopathic
hypersomnia were receiving CNS stimulants.
Abuse and Misuse
The active moiety of XYWAV is oxybate or gamma-hydroxybutyrate (GHB). Abuse or
misuse of illicit GHB, either alone or in combination with other CNS depressants, is
associated with CNS adverse reactions, including seizure, respiratory depression,
decreases in the level of consciousness, coma, and death.Because of the risks of CNS depression and abuse and misuse, XYWAV is available only
through a restricted program under a REMS called the XYWAV and XYREM REMS.Contraindications
XYWAV is contraindicatedin combination with sedative hypnotics or alcohol andin patients with succinic semialdehyde dehydrogenase deficiency.Warnings and Precautions
Central Nervous System Depression
The concurrent use of XYWAV with other CNS depressants, including but not limited to opioid analgesics, benzodiazepines, sedating antidepressants or antipsychotics, sedating anti-epileptic drugs, general anesthetics, muscle relaxants, and/or illicit CNS depressants, may increase the risk of respiratory depression, hypotension, profound sedation, syncope, and death. If use of these CNS depressants in combination with XYWAV is required, dose reduction or discontinuation of one or more CNS depressants (including XYWAV) should be considered. In addition, if short-term use of an opioid (e.g., post- or perioperative) is required, interruption of treatment with XYWAV should be considered.After first initiating treatment and until certain that XYWAV does not affect them adversely (e.g., impair judgment, thinking, or motor skills), caution patients against hazardous activities requiring complete mental alertness or motor coordination such as operating hazardous machinery, including automobiles or airplanes. Also caution patients against these hazardous activities for at least 6 hours after taking XYWAV. Patients should be queried about CNS depression-related events upon initiation of XYWAV therapy and
periodically thereafter.Abuse and Misuse
XYWAV is a Schedule Ill controlled substance. The active moiety of XYWAV is oxybate, also known as GHB, a Schedule I controlled substance. Abuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death. The rapid onset of sedation, coupled with the amnestic features of GHB particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (e.g., assault victim). Physicians should carefully evaluate patients for a history of drug abuse and follow such patients closely.XYWAV and XYREM REMS
Because of the risks of central nervous system depression and abuse and misuse, XYWAV is available only through a restricted distribution program called the XYWAV and XYREM REMS.Notable requirements of the XYWAV and XYREM REMS include the following:Healthcare Providers who prescribe XYWAV are specially certifiedXYWAV will be dispensed only by the central pharmacy that is specially certifiedXYWAV will be dispensed and shipped only to patients who are enrolled in the XYWAV and XYREM REMS with documentation of safe useFurther information is available at www.XYWAVXYREMREMS.com or 1-866-997-3688.Respiratory Depression and Sleep-Disordered Breathing
XYWAV may impair respiratory drive, especially in patients with compromised respiratory function. In overdoses of oxybate and with illicit use of GHB, life-threatening respiratory depression has been reported. Increased apnea and reduced oxygenation may occur with XYWAV administration in adult and pediatric patients. A significant increase in the number of central apneas and clinically significant oxygen desaturation may occur in patients with obstructive sleep apnea treated with XYWAV. Prescribers should be aware that sleep-related breathing disorders tend to be more prevalent in obese patients, in men, in postmenopausal women not on hormone replacement therapy, and among patients with narcolepsy.Depression and Suicidality
In Study 1, the randomized-withdrawal clinical trial in adult patients with narcolepsy (n=201), depression and depressed mood were reported in 3% and 4%, respectively, of patients treated with XYWAV. Two patients (1%) discontinued XYWAV because of depression. In most cases, no change in XYWAV treatment was required. In Study 2, the randomized-withdrawal clinical trial in adult patients with idiopathic hypersomnia (n=154), depression and depressed mood were reported in 1% and 3%, respectively, of patients treated with XYWAV. All patients continued XYWAV treatment.Two suicides and two attempted suicides occurred in adult clinical trials with oxybate (same active moiety as XYWAV). One patient experienced suicidal ideation and two patients reported depression in a pediatric clinical trial with oxybate. These events occurred in patients with and without previous histories of depressive disorders. The emergence of depression in patients treated with XYWAV requires careful and immediate evaluation. Monitor patients for the emergence of increased depressive symptoms and/or suicidality
while taking XYWAV.Other Behavioral or Psychiatric Adverse Reactions
In Study 1, confusion and anxiety occurred in 1% and 5% of patients with narcolepsy treated with XYWAV, respectively. One patient experienced visual hallucinations and confusion after ingesting approximately 9 grams of XYWAV.In Study 2, confusion and anxiety occurred in 3% and 16% of patients with idiopathic hypersomnia, respectively. One patient experienced visual hallucinations, which led to discontinuation of XYWAV. Other neuropsychiatric reactions reported with oxybate (same active moiety as XYWAV) in adult or pediatric clinical trials and in the postmarketing setting include hallucinations, paranoia, psychosis, aggression, agitation, confusion and anxiety. The emergence or increase in the occurrence of behavioral or psychiatric events in patients taking XYWAV should be carefully monitored.Parasomnias
Parasomnias can occur in patients taking XYWAV.In Study 1 and Study 2, parasomnias, including sleepwalking, were reported in 6% and 5% of adult patients treated with XYWAV, respectively. In a clinical trial of XYREM (same active moiety as XYWAV) in adult patients with narcolepsy, five instances of sleepwalking with potential injury or significant injury were reported. Parasomnias, including sleepwalking, have been reported in a pediatric clinical trial with sodium oxybate (same active moiety as XYWAV) and in postmarketing experience with sodium oxybate.Episodes of sleepwalking should be fully evaluated and appropriate interventions considered.Most Common Adverse Reactions
The most common adverse reactions (occurring in ≥5% of XYWAV-treated patients in adult clinical trials in either narcolepsy or IH) were nausea, headache, dizziness, anxiety, insomnia, decreased appetite, hyperhidrosis, vomiting, diarrhea, dry mouth, parasomnia, somnolence, fatigue, and tremor. In the pediatric clinical trial with XYREM (same active moiety as XYWAV) that included pediatric patients 7 to 17 years of age with narcolepsy, the most common adverse reactions (≥5%) were nausea (20%), enuresis (19%), vomiting (18%), headache (17%), weight decreased (13%), decreased appetite (9%), dizziness (8%), and sleepwalking (6%). The overall adverse reaction profile of XYREM in the pediatric clinical trial was similar to that seen in the adult clinical trial program. The safety profile in pediatric patients with XYWAV is expected to be similar to that of adult patients treated with XYWAV and to that of pediatric patients treated with XYREM.Additional Adverse Reactions
Adverse reactions that occurred in 2-
US Market News
4月前
Jazz Pharmaceuticals to Present Compelling Clinical and Pre-Clinical Data Advancing Oncology Research at AACR 2026March 18, 2026 7:44 AM
PR Newswire (US)
Seven presentations deliver new insights into zanidatamab's differentiated HER2 biology, emerging pre-clinical data on dordaviprone and related imipridones, and advancing research on JZP898, a conditionally activated IFN?2b cytokine For U.S. media and investors onlyDUBLIN, March 18, 2026 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the company and its partners will present one oral and six poster presentations at the 2026 American Association for Cancer Research (AACR) Annual Meeting, taking place April 17-22, 2026, in San Diego.The research highlights meaningful progress across Jazz's oncology portfolio, including new findings from company-sponsored research evaluating Ziihera® (zanidatamab-hrii), a HER2-targeted bispecific antibody; JZP898, an investigational, differentiated, conditionally activated interferon alpha-2b (IFN?2b) cytokine pro-drug designed for activation within the tumor microenvironment; and imipridone compounds including Modeyso™ (dordaviprone), a protease activator of the mitochondrial caseinolytic protease P (ClpP) and a dopamine D2 receptor (DRD2) inhibitor."Jazz is presenting research at AACR that highlights how we are advancing our oncology portfolio with pace and rigor to deliver differentiated science and new insights across multiple development programs," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "Zanidatamab is already delivering meaningful benefits for adult patients with previously treated, unresectable or metastatic HER2+ (IHC 3+) biliary tract cancer. We are advancing our program in first-line HER2+ locally advanced or metastatic gastroesophageal adenocarcinoma, and generating a robust body of evidence that characterizes its distinct HER2 biology and its potential across additional HER2-expressing tumors, including HER2+ breast cancer. The NeoZanHER trial data being presented at AACR provide supporting evidence for our ongoing research in early-stage breast cancer."Highlights at the AACR Annual Meeting include:An oral presentation with results from the Phase 2 single-arm, open-label NeoZanHER trial (NCT05035836) evaluating zanidatamab for the investigational use as neoadjuvant monotherapy in patients with early-stage HER2+ breast cancer. At six weeks, zanidatamab treatment resulted in a statistically significant decrease in tumor size and volume from baseline, and 30% of patients (n=6) achieved pathologic complete response (pCR). Treatment with zanidatamab was manageable with no new safety signals.A poster presentation detailing mechanistic and multi-omics analyses characterizing zanidatamab's differentiated HER2 biology, including dual, domain-specific binding and downstream effects on key cellular signaling pathways, with insights into activity in models following trastuzumab deruxtecan (T-DXd) exposure.A poster presentation featuring preclinical data supporting the activity of JZP898 – currently in Phase 1 development – including evidence of tumor-localized interferon signaling and immune engagement in preclinical models.A presentation featuring preclinical research evaluating imiprodones, inclusive of dordaviprone (formerly ONC201) and JZP3507 (formerly ONC206), across renal cell carcinoma and small-cell lung cancer models, including combination approaches and comparative analyses to further characterize activity and mechanism.Additional presentations will further explore zanidatamab's utility across HER2-expressing solid tumors and within innovative biomarker-driven clinical trial designs, including adaptive organ-preservation strategies in gastroesophageal adenocarcinoma (GEA).The AACR abstracts are available at: https://www.aacr.org/meeting/aacr-annual-meeting-2026/The full list of Jazz- and partner-supported presentations at the 2026 AACR Annual Meeting are:Zanidatamab Presentations:Presentation TitleAuthorsPresentation DetailsA phase 2 single-arm open-label trial evaluating zanidatamab in patients with early stage HER2 positive breast cancer: The NeoZanHER StudyValero V, Pohlmann PR, Mouabbi J, Huang X, Qiao W, Alonzo H, Murthy RK, Rauch GM, Adesoye T, Checka C, Symmans FW, Grachev D, Alajajyan F, Nwosu-Iheme A, Hassan A, Patel MM, Giordano SH, Hunt K, Tripathy D, Meric-Bernstam FType: Oral PresentationSession: Clinical Trials Minisymposium: Aiming for Cure: Perioperative Clinical TrialsDate/Time: April 18, 2026, 12:30-2:30 p.m. PST
Presentation number: CT012Zanidatamab modulates multiple pathways involved in tumor growth and survival and is efficacious post T-DXdKarmokar A, Loro E, Kyakulaga AH, Lau D, Weisser N, Desjardins G, Raghunatha P, Clark E, Humphreys R, and Vaidya KType: Poster
Session: Experimental and Molecular Therapeutics: Next-Generation Targeted Therapies Directed Against Tumor Surface Antigens
Date/Time: April 21, 2026, 9:00 a.m.-12:00 p.m. PSTAbstract number: 4542DiscovHER PAN-206: Phase 2 tumor-agnostic study of zanidatamab in patients with previously treated human epidermal growth factor receptor 2-overexpressing solid tumors Subbiah V, Makker V, Oh DY, Gardener E, Gartner E, Meric-Bernstam FType: Poster
Session: Phase II and Phase III Clinical Trials in ProgressDate/Time: April 21, 2026, 9:00 a.m.-12:00 p.m. PSTAbstract number: CT209AACR adaptive biomarker-driven organ preservation trial in GE adenocarcinomas (AACR-ADOPT-GEA)Elimova E, Ajani JA, Klempner SJ, Schalper K, Wainberg ZA, Yuan Y, Baranski JD, Boerner S, Durbin S, Gallagher D, Huh W, McLaughlin R, Strickland M, Rubin EH, Siu LL, Yap TA, LoRusso PType: PosterSession: Phase II and Phase III Clinical Trials in ProgressDate/Time: April 21, 2026, 9:00 a.m.-12:00 p.m. PST
Abstract number: CT223Dordaviprone (formerly ONC201) Presentations:Presentation TitleAuthorsPresentation DetailsNuvisertib (TP-3654) and Dordaviprone (ONC201) synergize to reduce renal cell carcinoma cell viabilityMeza KS, Holder SL, El-Deiry WType: Poster
Session: Experimental and Molecular Therapeutics: Targeting Cell Surface Vulnerabilities to Overcome Therapeutic Resistance
Date/Time: April 20, 2026, 2:00-5:00 p.m. PST
Abstract number: 3174Imipridones ONC201, ONC206, and ONC212 show potent killing and colony arrest of small-cell lung cancer cell linesSu AY, Purcell C, Zhang S, Zhou L, Uruchurtu AS, El-Deiry WSType: Poster
Session: Experimental and Molecular Therapeutics: Cellular Responses to Anticancer Drugs
Date/Time: April 20, 2026, 2:00-5:00 p.m. PSTAbstract number: 2937JZP898 Presentation:Presentation TitleAuthorsPresentation DetailsJZP898, a conditionally activated interferon alpha, generates efficacy and robust TME engagement in syngeneic mouse modelsKarmokar A, Loro E, Zhang Z, Mukavilli R, Gupta A, Trouba K, Amber V, Humphreys RCType: Poster
Session: Experimental and Molecular Therapeutics: RNA, Gene and Cell Therapies, and Enabling Assay Technologies
Date/Time: April 19, 2026, 2:00-5:00 p.m. PST
Abstract number: 470About Ziihera® (zanidatamab-hrii)
Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab-hrii induces complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC), and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.[1] In the United States, Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.1 The FDA granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).1 Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by?Jazz?and BeOne under license agreements from Zymeworks, which first developed the molecule.?A supplemental biologics license application for zanidatamab was submitted to the FDA under Real Time Oncology Review in first-line HER2+ locally advanced or metastatic GEA. The FDA granted two Breakthrough Therapy designations for zanidatamab's development: one as a single agent for previously treated HER2 gene-amplified BTC, and one in combination with standard-of-care chemotherapy for first-line HER2+ locally advanced or metastatic GEA. The FDA also granted two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for first-line GEA. Additionally, zanidatamab has received Orphan Drug designations from the FDA for the treatment of BTC, gastric (including gastroesophageal junction) cancer, and esophageal cancer, as well as Orphan Drug designations from the European Medicines Agency for the treatment of BTC, gastric/gastroesophageal junction cancer and oesophageal cancer. Important Safety Information for ZIIHERA WARNING: EMBRYO-FETAL?TOXICITY
Exposure to?ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patients
of the risk and need for effective contraception.WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity
ZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death. Verify the pregnancy status of females of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA. Left Ventricular Dysfunction
ZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to 2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA. The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%). USE IN SPECIFIC POPULATIONS Pediatric Use
Safety and efficacy of ZIIHERA have not been established in pediatric patients. Geriatric Use
Of the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older. No overall differences in safety or efficacy were observed between these patients and younger adult patients. The full U.S. Prescribing Information for ZIIHERA, including BOXED Warning, is available at: https://pp.jazzpharma.com/pi/ziihera.en.USPI.pdf About Modeyso™ (dordaviprone)
Modeyso (dordaviprone) is approved by FDA for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.[2] Modeyso is an orally administered small molecule given once weekly. Modeyso is a protease activator of the mitochondrial ClpP and also inhibits DRD2. In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma.2 Modeyso received accelerated approval based on a pre-specified integrated efficacy analysis of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). Continued approval may be contingent upon verification and description of clinical benefit in the ongoing Phase 3 ACTION trial (NCT05580562), which is evaluating the safety and clinical benefit of Modeyso in newly diagnosed patients with H3 K27M-mutant diffuse glioma following radiotherapy. The FDA granted dordaviprone Rare Pediatric Disease designation and Fast-Track designation, and dordaviprone received Orphan Drug Designation in the United States, Europe and Australia. Modeyso was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.Modeyso (dordaviprone) is not approved anywhere else in the world.IMPORTANT SAFETY INFORMATIONWARNINGS AND PRECAUTIONS
Hypersensitivity
MODEYSO can cause severe hypersensitivity reactions.In the pooled safety population, Grade 3 hypersensitivity reactions occurred in 0.3% of patients receiving MODEYSO. Signs and symptoms of hypersensitivity may include rash, hives, fever, low blood pressure, wheezing, or swelling of the face or throat.Inform patients about the signs and symptoms of hypersensitivity reactions and instruct them to seek immediate medical attention if symptoms occur.If clinically significant hypersensitivity or anaphylaxis occur, immediately interrupt MODEYSO and initiate appropriate medical treatment and supportive care. Based on the severity of the adverse reaction, temporarily interrupt or permanently discontinue MODEYSO.QTc Interval Prolongation
MODEYSO causes concentration-dependent QTc interval prolongation, which can increase the risk for ventricular tachyarrhythmias (e.g. torsades de pointes) or sudden death.In patients who received MODEYSO and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 6% and 1.2% of patients, respectively.Monitor ECGs and electrolytes prior to initiation and periodically during treatment, as clinically indicated. Increase the frequency of monitoring in patients with congenital long QT syndrome, existing QTc prolongation, a history of ventricular arrhythmias, electrolyte abnormalities, heart failure, or who are taking strong or moderate CYP3A4 inhibitors.Avoid concomitant use with other agents known to prolong the QT interval. If concomitant use cannot be avoided, increase the frequency of monitoring and separate administration of MODEYSO and QT-prolonging product.Interrupt or reduce the dose of MODEYSO in patients who develop QT prolongation; permanently discontinue in patients with signs of life-threatening arrhythmias.Embryo-Fetal Toxicity
MODEYSO can cause fetal harm when administered to a pregnant woman.Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose.ADVERSE REACTIONS
Serious adverse reactions occurred in 33% of the 376 patients who received MODEYSO. Serious adverse reactions in >2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%), and muscular weakness (2.1%). Fatal adverse reactions occurred in 1% of patients who received MODEYSO, including cardiac arrest (0.5%), intracranial hemorrhage (0.3%), and encephalopathy (0.3%).The most common adverse reactions (≥20%) reported in clinical trials with MODEYSO were fatigue (34%), headache (32%), vomiting (24%), nausea (24%), and musculoskeletal pain (20%). The most common (≥2%) Grade 3 or 4 laboratory abnormalities were decreased lymphocytes (7%), decreased calcium (2.7%), and increased alanine aminotransferase (2.4%).DRUG INTERACTIONS
Strong and Moderate CYP3A4 Inhibitors
Avoid concomitant use of MODEYSO with strong and moderate CYP3A4 inhibitors. If concomitant use cannot be avoided, reduce the MODEYSO dose as recommended and monitor for toxicity.Strong and Moderate CYP3A4 Inducers
Avoid concomitant use of strong and moderate CYP3A4 inducers with MODEYSO.USE IN SPECIFIC POPULATIONS
Lactation
There are no data on the presence of MODEYSO in human milk because of the potential for serious adverse reactions from MODEYSO in breastfed children, advise women not to breastfeed during treatment with MODEYSO and for 1 week after the last dose.Pediatric Use
The safety and effectiveness of MODEYSO have not been established in patients less than 1 year of age. Dosing has not been established for patients weighing less than 22 pounds (10 kg).Please refer to the full Prescribing Information, including both Patient Information and Instructions for Use, for complete safety and administration information.The full U.S. Prescribing Information for MODEYSO is available at: https://pp.jazzpharma.com/pi/modeyso.en.USPI.pdfAbout JZP898
JZP898 is an investigational differentiated, conditionally-activated IFNa INDUKINE™ molecule, and is currently in Phase 1 development for the investigational use in solid tumors as monotherapy and in combination with a PD-1 inhibitor. JZP898 is an engineered IFN?2b cytokine pro-drug that is activated specifically within the tumor microenvironment where it can stimulate IFNa receptors on cancer-fighting immune effector cells.About?Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with rare disease — often with limited or no therapeutic options. We have a diverse portfolio of medicines, including leading therapies addressing epilepsies, cancers and sleep disorders. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information.Cautionary Note Concerning Forward-Looking Statements
This press release contains forward-looking statements, including, but not limited to, statements related to the potential therapeutic benefits of zanidatamab in HER2+ first-line GEA and other HER2-expressing cancers, JZP898 and dordaviprone, and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the successful completion of regulatory activities and uncertain regulatory approval, risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals' Securities and Exchange Commission filings and reports, including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2025, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts:
Media Contact:
CorporateAffairsMediaInfo@jazzpharma.com
Ireland +353 1 637 2141
U.S. +1 215 867 4948Investor Contact:
InvestorInfo@jazzpharma.com
Ireland +353 1 634 3211
U.S. +1 650 496 27171 ZIIHERA (zanidatamab-hrii) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.)
2 MODEYSO (dordaviprone) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.
View original content to download multimedia:https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-to-present-compelling-clinical-and-pre-clinical-data-advancing-oncology-research-at-aacr-2026-302716697.htmlSOURCE Jazz Pharmaceuticals plc
Original: Jazz Pharmaceuticals to Present Compelling Clinical and Pre-Clinical Data Advancing Oncology Research at AACR 2026
UserAlias1
12年前
*DJ Jazz Pharmaceuticals: Phase 1 Clinical Data Generated To Date Supports Completing The Phase 1 Evaluation Of Jzp-386 At The Originally Planned Highest Dose
Last update: 09/12/2014 8:31:26 am
(MORE TO FOLLOW) Dow Jones Newswires (212-416-2800)
December 09, 2014 08:31 ET (13:31 GMT)
Copyright (c) 2014 Dow Jones & Company, Inc.
Jazz Pharma, Concert Pharma Report JZP-386 Program Update
Last update: 09/12/2014 8:30:50 am
Jazz Pharmaceuticals plc (NASDAQ: JAZZ) and Concert Pharmaceuticals, Inc.
(NASDAQ: CNCE) today announced that Phase 1 clinical data generated to date
supports completing the Phase 1 evaluation of JZP-386 at the originally
planned highest dose, which was not administered in the first Phase 1 trial
due to a technical dosing issue. The existing Phase 1 clinical data was
generated in a first-in-human trial evaluating the safety, pharmacokinetics,
and pharmacodynamics of JZP-386; enrollment was completed in the third
quarter. A second Phase 1 trial evaluating JZP-386 at the originally planned
highest dose is expected to be initiated in the first quarter of 2015, with
data expected in the second quarter of 2015.
JZP-386 is a deuterium-containing analog of sodium oxybate. The Phase 1
program is comparing JZP-386 to sodium oxybate versus placebo in healthy
volunteers. The companies expect that the results from the upcoming Phase 1
trial will inform the next steps in the development program for JZP-386.
Sodium oxybate is the active ingredient in Xyrem, a prescription medicine
marketed in the United States by Jazz Pharmaceuticals to treat cataplexy and
excessive daytime sleepiness in patients with narcolepsy, a serious
neurological disorder that affects approximately 1 in 2000 people in the
United States.
Jazz Pharmaceuticals and Concert Pharmaceuticals Provide JZP-386 Program Update
Last update: 09/12/2014 8:30:00 am
DUBLIN & LEXINGTON, Mass.--(BUSINESS WIRE)--December 09, 2014--
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) and Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) today announced that Phase 1 clinical data generated to date supports completing the Phase 1 evaluation of JZP-386 at the originally planned highest dose, which was not administered in the first Phase 1 trial due to a technical dosing issue. The existing Phase 1 clinical data was generated in a first-in-human trial evaluating the safety, pharmacokinetics, and pharmacodynamics of JZP-386; enrollment was completed in the third quarter. A second Phase 1 trial evaluating JZP-386 at the originally planned highest dose is expected to be initiated in the first quarter of 2015, with data expected in the second quarter of 2015.
JZP-386 is a deuterium-containing analog of sodium oxybate. The Phase 1 program is comparing JZP-386 to sodium oxybate versus placebo in healthy volunteers. The companies expect that the results from the upcoming Phase 1 trial will inform the next steps in the development program for JZP-386.
Sodium oxybate is the active ingredient in Xyrem(R), a prescription medicine marketed in the United States by Jazz Pharmaceuticals to treat cataplexy and excessive daytime sleepiness in patients with narcolepsy, a serious neurological disorder that affects approximately 1 in 2000 people in the United States.
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing and commercializing differentiated products that address unmet medical needs. The company has a diverse portfolio of products and/or product candidates in the areas of sleep, hematology/oncology, pain and psychiatry. The company's U.S. marketed products in these areas include: Xyrem(R) (sodium oxybate) oral solution, Erwinaze(R) (asparaginase Erwinia chrysanthemi), Prialt(R) (ziconotide) intrathecal infusion, Versacloz(R) (clozapine) oral suspension, FazaClo(R) (clozapine, USP) HD and FazaClo LD. Jazz Pharmaceuticals also has a number of products marketed outside the United States, including Erwinase(R) and Defitelio(R) (defibrotide). For more information, please visit www.jazzpharmaceuticals.com.
About Concert Pharmaceuticals
Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform(R) (deuterated chemical entity platform) to create novel small molecule drugs. This approach starts with approved drugs, advanced clinical candidates or previously studied compounds that have the potential to be improved with deuterium substitution to enhance clinical safety, tolerability and efficacy. The Company is developing a broad pipeline targeting CNS disorders, renal disease, inflammation and cancer. For more information, please visit www.concertpharma.com.
About Xyrem
Xyrem(R) (sodium oxybate) oral solution, CIII, is indicated for the treatment of cataplexy in narcolepsy and for the treatment of EDS in narcolepsy. Xyrem may only be dispensed to patients enrolled in the Xyrem Success Program(R). Xyrem was first approved in the United States in 2002. Safety and effectiveness in pediatric patients have not been established.
IMPORTANT SAFETY INFORMATION
------------------------------------------------------------------------------
XYREM is a Central Nervous System (CNS) depressant. In clinical trials at
recommended doses, obtundation and clinically significant respiratory
depression occurred in XYREM-treated patients. Almost all of the patients who
received XYREM during clinical trials in narcolepsy were receiving CNS
stimulants. XYREM is the sodium salt of gamma hydroxybutyrate (GHB). Abuse of
GHB, either alone or in combination with other CNS depressants, is associated
with CNS adverse reactions, including seizure, respiratory depression,
decreases in the level of consciousness, coma, and death. Because of the risks
of CNS depression, abuse, and misuse, XYREM is available only through a
restricted distribution program called the XYREM Success Program(R), using a
centralized pharmacy. Prescribers and patients must enroll in the program. For
further information go to www.XYREM.com or call 1-866-XYREM88(R)
(1-866-997-3688).
Xyrem is contraindicated in combination with sedative hypnotics or alcohol and in patients with succinic semialdehyde dehydrogenase deficiency. Use caution when considering the concurrent use of Xyrem with other CNS depressants. Healthcare providers should caution patients against hazardous activities requiring complete mental alertness or motor coordination within the first 6 hours of dosing or after first initiating treatment until certain that Xyrem does not affect them adversely. Xyrem is a Schedule III controlled substance. The rapid onset of sedation, coupled with the amnestic features of Xyrem, particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (e.g. assault victim). Monitor patients for emergent or increased depression and suicidality and for impaired motor/cognitive function. Episodes of sleepwalking should be fully evaluated and appropriate interventions considered. Consider the amount of daily sodium intake in each dose of Xyrem in patients sensitive to salt intake.
Jazz Pharmaceuticals plc "Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to future events in the Phase 1 clinical program for JZP-386, the timing of the second Phase 1 clinical trial, the therapeutic potential of JZP-386 and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the timing and conduct of clinical trials and the therapeutic value of JZP-386; the uncertainty of regulatory approval; and those risks with respect to research and development and clinical trials detailed from time-to-time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including the Quarterly Report on Form 10-Q for the quarter ended September 30, 2014, and future filings and reports by Jazz Pharmaceuticals. Jazz Pharmaceuticals undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.
Concert Pharmaceuticals Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including statements about the future clinical development of JZP-386, the potential effectiveness of JZP-386, our plans and timelines for the clinical development of JZP-386 and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals and other factors discussed in the " Risk Factors" section of our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.
Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.
Jazz Pharmaceuticals and the Jazz Pharmaceuticals logo are registered trademarks of Jazz Pharmaceuticals plc or its subsidiaries.
Photos/Multimedia Gallery Available: www.businesswire.com/multimedia/home/20141209005304/en/
CONTACT: Jazz Pharmaceuticals plc
(investors)
Katherine Littrell, PhD, RN, U.S. + 650-496-2717
Ireland + 353 1 634 7887
investorinfo@jazzpharma.com
or
(media)
Laurie Hurley, U.S. + 1-650-496-2796
Ireland + 353 1 634 7894
or
Concert Pharmaceuticals
(investors)
Justine Koenigsberg, U.S. + 781-674-5284
ir@concertpharma.com
or
(media)
The Yates Network
Kathryn Morris, U.S. + 845-635-9828
SOURCE: Jazz Pharmaceuticals plc and Concert Pharmaceuticals, Inc.
Copyright Business Wire 2014
(END) Dow Jones Newswires
December 09, 2014 08:30 ET (13:30 GMT)
UserAlias1
12年前
$JAZZ NEWS: Jazz Pharmaceuticals and Concert Pharmaceuticals Provide JZP-386 Program Update
Last update: 09/12/2014 8:30:00 am
DUBLIN & LEXINGTON, Mass.--(BUSINESS WIRE)--December 09, 2014--
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) and Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) today announced that Phase 1 clinical data generated to date supports completing the Phase 1 evaluation of JZP-386 at the originally planned highest dose, which was not administered in the first Phase 1 trial due to a technical dosing issue. The existing Phase 1 clinical data was generated in a first-in-human trial evaluating the safety, pharmacokinetics, and pharmacodynamics of JZP-386; enrollment was completed in the third quarter. A second Phase 1 trial evaluating JZP-386 at the originally planned highest dose is expected to be initiated in the first quarter of 2015, with data expected in the second quarter of 2015.
JZP-386 is a deuterium-containing analog of sodium oxybate. The Phase 1 program is comparing JZP-386 to sodium oxybate versus placebo in healthy volunteers. The companies expect that the results from the upcoming Phase 1 trial will inform the next steps in the development program for JZP-386.
Sodium oxybate is the active ingredient in Xyrem(R), a prescription medicine marketed in the United States by Jazz Pharmaceuticals to treat cataplexy and excessive daytime sleepiness in patients with narcolepsy, a serious neurological disorder that affects approximately 1 in 2000 people in the United States.
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing and commercializing differentiated products that address unmet medical needs. The company has a diverse portfolio of products and/or product candidates in the areas of sleep, hematology/oncology, pain and psychiatry. The company's U.S. marketed products in these areas include: Xyrem(R) (sodium oxybate) oral solution, Erwinaze(R) (asparaginase Erwinia chrysanthemi), Prialt(R) (ziconotide) intrathecal infusion, Versacloz(R) (clozapine) oral suspension, FazaClo(R) (clozapine, USP) HD and FazaClo LD. Jazz Pharmaceuticals also has a number of products marketed outside the United States, including Erwinase(R) and Defitelio(R) (defibrotide). For more information, please visit www.jazzpharmaceuticals.com.
About Concert Pharmaceuticals
Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform(R) (deuterated chemical entity platform) to create novel small molecule drugs. This approach starts with approved drugs, advanced clinical candidates or previously studied compounds that have the potential to be improved with deuterium substitution to enhance clinical safety, tolerability and efficacy. The Company is developing a broad pipeline targeting CNS disorders, renal disease, inflammation and cancer. For more information, please visit www.concertpharma.com.
About Xyrem
Xyrem(R) (sodium oxybate) oral solution, CIII, is indicated for the treatment of cataplexy in narcolepsy and for the treatment of EDS in narcolepsy. Xyrem may only be dispensed to patients enrolled in the Xyrem Success Program(R). Xyrem was first approved in the United States in 2002. Safety and effectiveness in pediatric patients have not been established.
IMPORTANT SAFETY INFORMATION
------------------------------------------------------------------------------
XYREM is a Central Nervous System (CNS) depressant. In clinical trials at
recommended doses, obtundation and clinically significant respiratory
depression occurred in XYREM-treated patients. Almost all of the patients who
received XYREM during clinical trials in narcolepsy were receiving CNS
stimulants. XYREM is the sodium salt of gamma hydroxybutyrate (GHB). Abuse of
GHB, either alone or in combination with other CNS depressants, is associated
with CNS adverse reactions, including seizure, respiratory depression,
decreases in the level of consciousness, coma, and death. Because of the risks
of CNS depression, abuse, and misuse, XYREM is available only through a
restricted distribution program called the XYREM Success Program(R), using a
centralized pharmacy. Prescribers and patients must enroll in the program. For
further information go to www.XYREM.com or call 1-866-XYREM88(R)
(1-866-997-3688).
Xyrem is contraindicated in combination with sedative hypnotics or alcohol and in patients with succinic semialdehyde dehydrogenase deficiency. Use caution when considering the concurrent use of Xyrem with other CNS depressants. Healthcare providers should caution patients against hazardous activities requiring complete mental alertness or motor coordination within the first 6 hours of dosing or after first initiating treatment until certain that Xyrem does not affect them adversely. Xyrem is a Schedule III controlled substance. The rapid onset of sedation, coupled with the amnestic features of Xyrem, particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (e.g. assault victim). Monitor patients for emergent or increased depression and suicidality and for impaired motor/cognitive function. Episodes of sleepwalking should be fully evaluated and appropriate interventions considered. Consider the amount of daily sodium intake in each dose of Xyrem in patients sensitive to salt intake.
Jazz Pharmaceuticals plc "Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to future events in the Phase 1 clinical program for JZP-386, the timing of the second Phase 1 clinical trial, the therapeutic potential of JZP-386 and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the timing and conduct of clinical trials and the therapeutic value of JZP-386; the uncertainty of regulatory approval; and those risks with respect to research and development and clinical trials detailed from time-to-time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including the Quarterly Report on Form 10-Q for the quarter ended September 30, 2014, and future filings and reports by Jazz Pharmaceuticals. Jazz Pharmaceuticals undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.
Concert Pharmaceuticals Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including statements about the future clinical development of JZP-386, the potential effectiveness of JZP-386, our plans and timelines for the clinical development of JZP-386 and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals and other factors discussed in the " Risk Factors" section of our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.
Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.
Jazz Pharmaceuticals and the Jazz Pharmaceuticals logo are registered trademarks of Jazz Pharmaceuticals plc or its subsidiaries.
Photos/Multimedia Gallery Available: www.businesswire.com/multimedia/home/20141209005304/en/
CONTACT: Jazz Pharmaceuticals plc
(investors)
Katherine Littrell, PhD, RN, U.S. + 650-496-2717
Ireland + 353 1 634 7887
investorinfo@jazzpharma.com
or
(media)
Laurie Hurley, U.S. + 1-650-496-2796
Ireland + 353 1 634 7894
or
Concert Pharmaceuticals
(investors)
Justine Koenigsberg, U.S. + 781-674-5284
ir@concertpharma.com
or
(media)
The Yates Network
Kathryn Morris, U.S. + 845-635-9828
SOURCE: Jazz Pharmaceuticals plc and Concert Pharmaceuticals, Inc.
Copyright Business Wire 2014
(END) Dow Jones Newswires
December 09, 2014 08:30 ET (13:30 GMT)
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