Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical
company focused on transforming medicines to transform lives, today
announced the publication of final data from the RESTORE
open-label/switch study, demonstrating that people with narcolepsy
prefer once-at-bedtime LUMRYZ versus a twice-nightly
immediate-release oxybate treatment option. The U.S. Food and Drug
Administration approved LUMRYZ in 2023 as the first and only
once-nightly oxybate, which marked a significant milestone for
people with narcolepsy, as twice-nightly oxybates were the only
other oxybate options available for more than 20 years.
The paper, titled “RESTORE: Once-Nightly Oxybate Dosing
Preference and Nocturnal Experience With Twice-Nightly Oxybates,”
was published online in Sleep Medicine: X. Initial and end-of-study
survey data from RESTORE were previously presented in April 2022 at
the American Academy of Neurology Annual Meeting and in June 2024
at SLEEP 2024.
“Twice-nightly oxybates can present significant treatment
burdens and adherence concerns given the required
middle-of-the-night awakening for a second dose. RESTORE study
participants overwhelmingly preferred the once-nightly dosing
regimen of LUMRYZ over twice-nightly oxybates. RESTORE further
highlighted the inconvenience with the middle-of-the-night dose, as
well as the associated grogginess and unsteadiness the following
morning if the dose was taken late, and the worsening of symptoms
when the dose was missed,” said Asim Roy, M.D., co-author of the
paper and RESTORE investigator, and Medical Director of the Ohio
Sleep Medicine Institute. “With LUMRYZ, the majority of switch
participants who participated in the survey reported improvements
in their ability to get through the day without falling asleep,
being better able to sleep through the night, and accomplish more
in professional and social settings.”
As part of the multicenter, open-label Phase 3 RESTORE
extension/switch study, participants with narcolepsy type 1 or 2
who switched from a twice-nightly oxybate to once-nightly LUMRYZ
completed a nocturnal adverse events questionnaire at baseline, a
patient preference questionnaire after three months of taking
LUMRYZ, and an end-of-study questionnaire.
- Of the 129 switch participants who completed the nocturnal
adverse events questionnaire at baseline:
- 69% reported missing their second oxybate dose, and of those
participants, 80% felt that control of their symptoms was worse the
next day compared to days after which they had taken both doses as
prescribed
- More than half (51%) of the 51 participants who took their
second nightly oxybate dose more than four hours after the first
dose reported feeling somewhat to extremely groggy or unsteady the
next morning
- 92% reported getting out of bed after taking their second dose
of oxybate, with 7.5% of those reporting falling after waking up
for the second dose, and 4.2% reporting injuries
- 23% of participants stated they required another person to wake
with them in the middle of the night to ensure they took the second
dose of their twice-nightly oxybate
- Of the 98 switch participants who completed the patient
preference questionnaire, 94% preferred once-at-bedtime LUMRYZ to
twice-nightly oxybate dosing
- Of the 68 switch participants who completed the end-of-study
questionnaire:
- 79% were very satisfied with LUMRYZ compared to other
narcolepsy treatments they had previously taken
- 93% would recommend LUMRYZ to a family member or friend with
narcolepsy
- 91% said they were better able to sleep through the night since
starting treatment with LUMRYZ
- 91% said they were better able to follow the recommended
medication schedule of LUMRYZ than their previous oxybate
“The RESTORE study, which was conducted for more than three
years, allowed investigators switching participants from
twice-nightly, first-generation oxybates to understand the myriad
challenges associated with chronically taking a middle-of-the-night
dose of medication. Nearly 1 out of 4 switch participants reported
needing someone else to wake up with them to take the
middle-of-the-night dose. Once-at-bedtime LUMRYZ avoids the dosing
burden of a twice-nightly oxybate, which as RESTORE affirms, is
experienced in multiple negative aspects for patients, which can be
avoided with LUMRYZ. Not surprisingly, among those switching, more
than nine in 10 would recommend LUMRYZ to a family member or friend
with narcolepsy,” said Jennifer Gudeman, Pharm.D., Senior Vice
President, Medical and Clinical Affairs of Avadel. “Data from
RESTORE demonstrate LUMRYZ was well tolerated, with a low rate of
discontinuation due to adverse events, and further confirmed the
long-term safety and tolerability of this innovative treatment
option.”
About LUMRYZ™ (sodium oxybate) for extended-release
oral suspensionLUMRYZ is an extended-release sodium
oxybate medication approved by the FDA on May 1, 2023, as the
first and only once-at-bedtime treatment for cataplexy or excessive
daytime sleepiness (EDS) in adults with narcolepsy.
The FDA approval of LUMRYZ was supported by results from
REST-ON, a randomized, double-blind, placebo-controlled, pivotal
Phase 3 trial in adults with narcolepsy. LUMRYZ demonstrated
statistically significant and clinically meaningful improvements in
the three co-primary endpoints: EDS, clinicians’ overall assessment
of patients’ functioning (CGI-I), and cataplexy attacks, for all
three evaluated doses when compared to placebo.
With its approval, the FDA also granted seven years of Orphan
Drug Exclusivity to LUMRYZ for the treatment of cataplexy or EDS in
adults with narcolepsy due to a finding of clinical superiority of
LUMRYZ relative to currently available oxybate treatments. In
particular, the FDA found that LUMRYZ makes a major contribution to
patient care over currently available, twice-nightly oxybate
products by providing a once-nightly dosing regimen that avoids
nocturnal arousal to take a second dose.
IMPORTANT SAFETY INFORMATION
WARNING: Taking LUMRYZ™ (sodium oxybate) with other central
nervous system (CNS) depressants, such as medicines used to make
you fall asleep, including opioid analgesics, benzodiazepines,
sedating antidepressants, antipsychotics, sedating anti-epileptic
medicines, general anesthetics, muscle relaxants, alcohol or street
drugs, may cause serious medical problems, including trouble
breathing (respiratory depression), low blood pressure
(hypotension), changes in alertness (drowsiness), fainting
(syncope) and death. |
|
The active ingredient of LUMRYZ (sodium oxybate) is a form
of gamma hydroxybutyrate (GHB), a controlled substance. Abuse or
misuse of illegal GHB alone or with other CNS depressants (drugs
that cause changes in alertness or consciousness) have caused
serious side effects. These effects include seizures, trouble
breathing (respiratory depression), changes in alertness
(drowsiness), coma and death. Call your doctor right away if you
have any of these serious side effects. |
|
Because of these risks, LUMRYZ is available only by
prescription and filled through certified pharmacies in the LUMRYZ
REMS. You must be enrolled in the LUMRYZ REMS to receive LUMRYZ.
Further information is available at www.LUMRYZREMS.com or by
calling 1-877-453-1029. |
|
INDICATIONSLUMRYZ (sodium oxybate) for
extended-release oral suspension is a prescription medicine used to
treat the following symptoms in adults with narcolepsy:
- sudden onset of weak or paralyzed muscles (cataplexy)
- excessive daytime sleepiness (EDS)
It is not known if LUMRYZ is safe and effective in people less
than 18 years of age.
Do not take LUMRYZ if you take other sleep
medicines or sedatives (medicines that cause sleepiness), drink
alcohol or have a rare problem called succinic semialdehyde
dehydrogenase deficiency.
Keep LUMRYZ in a safe place to prevent abuse and misuse. Selling
or giving away LUMRYZ may harm others and is against the law. Tell
your doctor if you have ever abused or been dependent on alcohol,
prescription medicines or street drugs.
Anyone who takes LUMRYZ should not do anything that requires
them to be fully awake or is dangerous, including driving a car,
using heavy machinery or flying an airplane, for at least six (6)
hours after taking LUMRYZ. Those activities should not be done
until you know how LUMRYZ affects you.
Falling asleep quickly, including while standing or while
getting up from the bed, has led to falls with injuries that have
required some people to be hospitalized.
LUMRYZ can cause serious side effects, including the
following:
- Breathing problems, including slower
breathing, trouble breathing and/or short periods of not breathing
while sleeping (e.g., sleep apnea). People who already have
breathing or lung problems have a higher chance of having breathing
problems when they take LUMRYZ.
- Mental health problems,
including confusion, seeing or hearing things that are not
real (hallucinations), unusual or disturbing thoughts (abnormal
thinking), feeling anxious or upset, depression, thoughts of
killing yourself or trying to kill yourself, increased tiredness,
feelings of guilt or worthlessness and difficulty concentrating.
Tell your doctor if you have or had depression or have tried to
harm yourself. Call your doctor right away if you have
symptoms of mental health problems or a change in weight or
appetite.
- Sleepwalking. Sleepwalking can cause
injuries. Call your doctor if you start sleepwalking.
Tell your doctor if you are on a salt-restricted diet or if you
have high blood pressure, heart failure or kidney problems. LUMRYZ
contains a lot of sodium (salt) and may not be right for you.
The most common side effects of LUMRYZ in adults include nausea,
dizziness, bedwetting, headache and vomiting. Your side effects may
increase when you take higher doses of LUMRYZ. LUMRYZ can cause
physical dependence and craving for the medicine when it is not
taken as directed. These are not all the possible side effects of
LUMRYZ.
For more information, ask your doctor or pharmacist.
Call your doctor for medical advice about side
effects.
You are encouraged to report negative side effects of
prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or
call 1-800-FDA-1088.
Please see full Prescribing Information, including
BOXED Warning.
About Avadel Pharmaceuticals plcAvadel
Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company
focused on transforming medicines to transform lives. Our approach
includes applying innovative solutions to the development of
medications that address the challenges patients face with current
treatment options. Avadel’s commercial product, LUMRYZ™, was
approved by the U.S. Food & Drug Administration (FDA)
as the first and only once-at-bedtime oxybate for the treatment of
cataplexy or excessive daytime sleepiness (EDS) in adults with
narcolepsy. For more information, please
visit www.avadel.com.
Cautionary Disclosure Regarding Forward-Looking
StatementsThis press release includes “forward-looking
statements” within the meaning of Section 27A of the Securities Act
of 1933 and Section 21E of the Securities Exchange Act of 1934.
These forward-looking statements relate to our future expectations,
beliefs, plans, strategies, objectives, results, conditions,
financial performance, prospects or other events. Such
forward-looking statements include, but are not limited to,
expectations regarding the potential therapeutic benefit of LUMRYZ
in treating narcolepsy-related EDS or cataplexy, including
potential impact on patient weight; and the results and analysis of
the Phase 3 REST-ON trial, including the details and content
thereof. In some cases, forward-looking statements can be
identified by the use of words such as “will,” “may,” “could,”
“believe,” “expect,” “look forward,” “on track,” “guidance,”
“anticipate,” “estimate,” “project,” “next steps” and similar
expressions and the negatives thereof (if applicable).
The Company’s forward-looking statements are based on estimates
and assumptions that are made within the bounds of our knowledge of
our business and operations and that we consider reasonable.
However, the Company’s business and operations are subject to
significant risks, and, as a result, there can be no assurance that
actual results and the results of the company’s business and
operations will not differ materially from the results contemplated
in such forward-looking statements. Factors that could cause actual
results to differ from expectations in the Company’s
forward-looking statements include the risks and uncertainties
described in the “Risk Factors” section of Part I, Item 1A of the
Company’s Annual Report on Form 10-K for the year ended December
31, 2023, which was filed with the Securities and Exchange
Commission (SEC) on February 29, 2024, and subsequent SEC filings.
Forward-looking statements speak only as of the date they are made
and are not guarantees of future performance. Accordingly, you
should not place undue reliance on forward-looking statements. The
Company does not undertake any obligation to publicly update or
revise our forward-looking statements, except as required by
law.
Investor Contact:Courtney MogerleyPrecision
AQCourtney.Mogerley@precisionAQ.com(212)
698-8687
Media Contact:Lesley StanleyReal
Chemistrylestanley@realchemistry.com(609)
273-3162
Avadel Pharmaceuticals (NASDAQ:AVDL)
過去 株価チャート
から 8 2024 まで 9 2024
Avadel Pharmaceuticals (NASDAQ:AVDL)
過去 株価チャート
から 9 2023 まで 9 2024