Vertex Pharmaceuticals Inc【VRTX】株式ニュース

Vertex Announces US FDA Approval for Label Extensions of ALYFTREK® and TRIKAFTA®, Expanding Availability of These Medicines to ~95% of All People With CF in the United StatesApril 1, 2026 7:30 AM
Business Wire
-With this expansion, any variant that results in production of CFTR protein is now included in the indication for ALYFTREK and TRIKAFTA, reinforcing the impact these medicines have, regardless of the location of the variant in the CFTR protein-

-Approximately 800 more people with CF in the US are now eligible for a CFTR modulator for the first time-

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved expanded use of ALYFTREK® (vanzacaftor/tezacaftor/ivacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 and older with a variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. Additionally, the U.S. FDA has also expanded the indication statement for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in patients ages 2 and older.

This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to ALYFTREK and 521 variants demonstrating response to TRIKAFTA. As such, approximately 800 more people with a clinical diagnosis of CF in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time. This extension means approximately 95% of people with CF in the U.S. are now eligible for treatment with a CFTR modulator.

“This groundbreaking approval represents more than 20 years of innovation in CF, including testing over 600 variants in our laboratory, demonstrating clinical effects in large clinical trials, and studying younger people with CF so they can be treated as early as possible,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “With this label expansion, any variant that results in production of CFTR protein is now included in the ALYFTREK and TRIKAFTA labels, validating that these medicines can restore CFTR function and provide clinical benefit to patients regardless of where in the CFTR protein a variant is located. We thank the CF community and investigators for their trust and look forward to bringing ALYFTREK and TRIKAFTA to more patients than ever before.”

For more information on ALYFTREK or TRIKAFTA, patient assistance programs, or eligibility details, visit ALYFTREK.com, TRIKAFTA.com, VertexGPS.com or vertextreatments.com.

IMPORTANT SAFETY INFORMATION

WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE

Elevated transaminases have been observed in patients treated with ALYFTREK.

TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. Cases of liver failure leading to transplantation and death have been reported in both clinical trials and the postmarketing setting in patients with and without a history of liver disease taking TRIKAFTA, a fixed-dose combination drug containing elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA), the same or similar active ingredients as ALYFTREK. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA.

Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or liver function test (LFT) elevations at baseline.

Interrupt ALYFTREK or TRIKAFTA for significant elevations in LFTs or in the event of signs or symptoms of liver injury. Consider referral to a hepatologist. Follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, resume treatment only if benefit is expected to outweigh risk. Closer monitoring is advised after resuming treatment.

ALYFTREK or TRIKAFTA should not be used in patients with severe hepatic impairment (Child-Pugh Class C). ALYFTREK or TRIKAFTA is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B). ALYFTREK or TRIKAFTA should only be considered when there is a clear medical need and benefit outweighs risk. If ALYFTREK is used, monitor patients closely. If TRIKAFTA is used, use with caution at a reduced dosage and monitor patients closely.

WARNINGS AND PRECAUTIONS

DRUG-INDUCED LIVER INJURY AND LIVER FAILURE

Elevated transaminases have been observed in patients treated with ALYFTREK. TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. Liver failure leading to transplantation and death has been reported in patients with and without a history of liver disease taking TRIKAFTA. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA

Assess LFTs in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or LFT elevations at baseline, or a history of elevated LFTs with drugs containing ELX, TEZ, and/or IVA

Interrupt ALYFTREK or TRIKAFTA in the event of signs or symptoms of liver injury, which may include:

Significant elevations in LFTs (e.g., ALT or AST >5x the upper limit of normal (ULN) or ALT or AST >3x ULN with bilirubin >2x ULN)

Clinical symptoms suggestive of liver injury (e.g., jaundice, right upper quadrant pain, nausea, vomiting, altered mental status, ascites)

Consider referral to a hepatologist and follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, and if benefit is expected to outweigh risk, resume treatment with close monitoring

ALYFTREK and TRIKAFTA should not be used in patients with severe hepatic impairment, are not recommended in patients with moderate hepatic impairment, and should only be considered when there is a clear medical need and benefit outweighs risk. If ALYFTREK is used, monitor patients closely. If TRIKAFTA is used, use with caution at a reduced dosage and monitor patients closely

HYPERSENSITIVITY REACTIONS, INCLUDING ANAPHYLAXIS

Hypersensitivity reactions, including cases of angioedema and anaphylaxis, have been reported in the postmarketing setting for TRIKAFTA. If signs or symptoms of serious hypersensitivity reactions develop during treatment, discontinue ALYFTREK or TRIKAFTA and institute appropriate therapy. Consider benefits and risks to determine whether to resume treatment

PATIENTS WHO DISCONTINUED OR INTERRUPTED ELX-, TEZ-, OR IVA-CONTAINING DRUGS DUE TO ADVERSE REACTIONS

ALYFTREK

There are no available safety data for ALYFTREK in patients who previously discontinued or interrupted treatment with drugs containing ELX, TEZ, or IVA due to adverse reactions. Consider benefits and risks before using ALYFTREK in these patients and if used, closely monitor for adverse reactions

INTRACRANIAL HYPERTENSION (IH)

IH has been reported in the postmarketing setting with TRIKAFTA, which contains the same or similar active ingredients as ALYFTREK. Clinical manifestations of IH include headache, blurred vision, diplopia, and potential vision loss; papilledema can be found on fundoscopy. If an unusual headache or visual disturbances occur during treatment, and IH is suspected, interrupt treatment and refer for prompt medical evaluation. Consider benefits and risks to determine whether to resume treatment. Patients should be monitored until IH resolution and for recurrence. Patients with elevated vitamin A levels may be at increased risk

NEUROPSYCHIATRIC EVENTS, INCLUDING SUICIDAL THOUGHTS AND BEHAVIORS

Serious neuropsychiatric events, including symptoms of anxiety, depression, suicidal ideation and behavior, and sleep disturbances, have been reported in the postmarketing setting in patients with and without a previous history of neuropsychiatric symptoms taking ALYFTREK or TRIKAFTA. Symptoms may occur within the first 3 months of treatment. Assess patients for baseline neuropsychiatric symptoms and monitor for new or worsening symptoms. Consider the benefits and risks to determine if treatment should be interrupted at symptom occurrence or resumed with symptom improvement

DRUG INTERACTIONS

Use With CYP3A Inducers

Following concomitant use of strong or moderate CYP3A inducers with ALYFTREK, exposures of vanzacaftor, TEZ, and deutivacaftor were decreased, which may reduce ALYFTREK effectiveness. Concomitant use with strong or moderate CYP3A inducers is not recommended

Exposure to IVA is significantly decreased and exposure to ELX and TEZ are expected to decrease with concomitant use of CYP3A inducers, which may reduce effectiveness of TRIKAFTA. Concomitant use with strong CYP3A inducers is not recommended

Use With CYP3A Inhibitors

Exposure to vanzacaftor, TEZ, and deutivacaftor or ELX, TEZ, and IVA are increased when used concomitantly with strong or moderate CYP3A inhibitors. The dose of ALYFTREK or TRIKAFTA should be reduced when used concomitantly with moderate or strong CYP3A inhibitors

CATARACTS

Non-congenital lens opacities have been reported in pediatric patients treated with TRIKAFTA, which contains IVA (similar to an active ingredient in ALYFTREK). Baseline and follow-up ophthalmological examinations are recommended in pediatric patients

ADVERSE REACTIONS

ALYFTREK

Serious adverse reactions that occurred more frequently with ALYFTREK than with ELX/TEZ/IVA in 2 or more patients (≥0.4%) were influenza (1.5%), increased AST (0.4%), increased GGT (0.4%), depression (0.4%), and syncope (0.4%)

The most common adverse reactions occurring in ≥5% of patients and at a frequency higher than ELX/TEZ/IVA by ≥1% were cough, nasopharyngitis, upper respiratory tract infection (URTI), headache, oropharyngeal pain, influenza, fatigue, increased ALT and AST, rash, and sinus congestion

TRIKAFTA

Serious adverse reactions that occurred more frequently in patients treated with TRIKAFTA compared to placebo included rash (1% vs

Breaking Barriers: How 2026's Top Clinical Leaders Are Disrupting Chronic Disease MarketsJanuary 26, 2026 3:16 PM
PR Newswire (US)

Issued on behalf of Avant Technologies Inc.Equity Insider News CommentaryVANCOUVER, BC, Jan. 26, 2026 /PRNewswire/ -- The medical world is shifting as the global market for next-gen treatments heads toward $88.85 billion by 2030[1], driven by a surge in funding for high-tech cures for long-term illness. A major FDA move at the beginning of the year[2] is now making it much easier to build these advanced platforms, clearing a path for companies that can treat diseases internally without the harsh side effects of traditional drugs. This regulatory shift positions Avant Technologies, Inc. (OTCQB: AVAI), MannKind (NASAQ: MNKD), Vertex Pharmaceuticals (NASDAQ: VRTX), Fate Therapeutics (NASDAQ: FATE), and Ardelyx (NADSAQ: ARDX) at the center of a massive transformation in chronic healthcare. Smart investors are preparing for a major 2026 rebound as capital flows into the most innovative and results-driven medical platforms[3]. With the total biotech sector projected to reach $9.06 trillion by 2035[4], the biggest opportunities are in companies solving massive issues like diabetes and kidney disease with better clinical execution. In 2026, the market is favoring 'ready-to-go' platforms over early-stage speculation, rewarding the companies that have built the infrastructure to dominate the next generation of medicine.Avant Technologies, Inc. (OTCQB: AVAI) is tackling some of healthcare's biggest challenges with a novel approach: genetically modified cells that produce therapeutic proteins inside the body, protected by a proprietary shield that keeps the immune system from attacking them. The company operates through two joint ventures targeting markets worth hundreds of billions of dollars.The core innovation is a cell encapsulation technology that solves a fundamental problem in regenerative medicine. When doctors transplant therapeutic cells into patients, the immune system typically destroys them within days or weeks. The traditional solution requires lifelong immunosuppressive drugs that cause serious side effects including infection risk, organ damage, and elevated cancer risk. Avant's technology eliminates this problem by creating a protective barrier around the cells while still allowing nutrients, oxygen, and therapeutic proteins to pass through freely.The first venture, Insulinova, Inc., partners with SGAustria Pte. Ltd. to develop treatments for type 1 diabetes and insulin-dependent type 2 diabetes. The approach uses genetically modified cells that produce, regulate and store insulin, essentially creating a bio artificial pancreas that restores natural glucose control without immunosuppressive drugs. The diabetes market opportunity is substantial: 589 million people globally live with type 1 and insulin-dependent type 2 diabetes, projected to reach 853 million by 2050 according to the International Diabetes Federation."Cell encapsulation is a game changer in the field of regenerative medicine," said Chris Winter, CEO of Avant Technologies. "By partnering with SGAustria, we're ensuring that genetically modified insulin-producing cells can thrive in the body long-term and offer the potential of restoring natural glucose control and dramatically improving patients' quality of life. This technology not only minimizes risks like immune rejection but also prevents potential complications such as cell escape or tumor formation, making it a cornerstone for safe and scalable diabetes therapies."Avant's second venture, Klothonova, partners with Singapore-based Austrianova to develop therapies for both age-related diseases and anti-aging therapies using cells that produce the Klotho protein. Research from the Mayo Clinic links declining Klotho levels to arterial stiffness, endothelial dysfunction, and vascular calcification.Both platforms are backed by over 50 peer-reviewed publications representing decades of development. The addressable markets span multiple areas: Alzheimer's disease ($32.8 billion by 2033), cardiovascular disease (32% of global deaths), and kidney disease (850 million affected worldwide).The strategic advantage lies in platform versatility. The same encapsulation technology that protects insulin-producing cells can theoretically protect cells producing other therapeutic proteins, potentially opening pathways into additional disease indications. This positions Avant at the intersection of multiple high-value healthcare markets with a single core technology that addresses the immune rejection challenge across different applications.CONTINUED... Read this and more news for Avant Technologies at:
https://equity-insider.com/2025/03/21/unlocking-the-trillion-dollar-ai-market-what-investors-need-to-know/ MannKind (NASDAQ: MNKD) provided business updates outlining anticipated growth drivers for 2026, including progress across commercial programs and clinical development initiatives following a record-setting fourth quarter surpassing $100 million in net revenue. The company expects two high-potential launches on the horizon with FDA decisions anticipated for Afrezza label updates and FUROSCIX ReadyFlow Autoinjector."MannKind closed 2025 on a high note, marked by milestones that reinforce our growth trajectory—including the acquisition of scPharmaceuticals and a record-setting fourth quarter surpassing $100 million in net revenue," said Michael Castagna, PharmD, CEO of MannKind Corporation. "With two high-potential launches on the horizon, 2026 is shaping up to be a catalyst-rich year that positions MannKind for long-term value creation."Major catalysts include an FDA decision on Afrezza dose conversion with PDUFA date of January 23, 2026, and review of the supplemental BLA for Afrezza in pediatric patients with PDUFA date of May 29, 2026. MannKind also continues advancing its pipeline including nintedanib DPI with first patient enrollment in the INFLO-1 Phase 1b study completed in December.Vertex Pharmaceuticals (NASDAQ: VRTX) provided pipeline and business updates ahead of the J.P. Morgan Healthcare Conference, highlighting strong commercial execution and rapid R&D progress setting up continued growth and important milestones for 2026. The company increased estimates for people with CF in all target markets to approximately 112,000 including approximately 97,000 in core markets, while CASGEVY realized greater than $100 million revenue in 2025 reflecting more than 60 patient infusions."2025 was a year of strong commercial execution and rapid R&D progress, setting up the company for continued growth and many important milestones in 2026," said Reshma Kewalramani, M.D., CEO and President of Vertex. "Building on this momentum, we are focused on expanding our commercial reach in multiple disease areas; advancing the emerging renal franchise, including the potential near-term launch of povetacicept; and progressing our mid- and late-stage clinical pipeline."Vertex expects to complete the rolling BLA filing for U.S. accelerated approval of povetacicept in IgAN in the first half of 2026 using a priority review voucher to expedite review from ten months to six months. The company also plans to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy by end of 2026, with more than 500,000 JOURNAVX prescriptions written and filled in 2025.Fate Therapeutics (NASDAQ: FATE) presented updated Phase 1 clinical data of FT819 off-the-shelf CAR T-cell product candidate demonstrating meaningful decrease in disease and favorable safety profile with twelve systemic lupus erythematosus patients now treated and first systemic sclerosis patient dosed. The company continues to advance preparations for a pivotal study and is engaged in discussions with the FDA under its RMAT designation regarding plans to initiate registrational trial in 2026."We are very pleased with the accelerating patient enrollment, the expansion of U.S. clinical sites, and the addition of international clinical sites, which together are enabling broader access to FT819 for patients suffering with lupus," said Bob Valamehr, Ph.D., M.B.A., President and CEO of Fate Therapeutics. "The updated FT819 clinical data continue to demonstrate meaningful and durable responses with the use of less-intensive conditioning chemotherapy and a differentiated safety profile."Preliminary data in Regimen A showed mean SLEDAI-2K score decreased progressively from baseline with DL1 dropping 50% at month 3 and 70% at month 6, while DL2 decreased 65% at month 3 and 78% at month 6. Clinical SLEDAI-2K of 0 was achieved in 5 out of 10 patients with no Grade >2 CRS, ICANS, or GVHD reported.Ardelyx (NASDAQ: ARDX) presented real-world evidence studies of XPHOZAH (tenapanor) demonstrating patient satisfaction and reduction in serum phosphate at the American Society of Nephrology's Kidney Week. The first real-world study showed patients prescribed tenapanor experienced a reduction in serum phosphate of nearly 1 mg/dL on average, with 45.3% experiencing at least 1 mg/dL reduction."We are excited to present new data on XPHOZAH at ASN's Kidney Week, including the first results from our prospective, observational cohort study designed to evaluate the impact of an XPHOZAH-based regimen in a real-world setting," said Edward Conner, Chief Medical Officer. "Our results show the impact XPHOZAH can have in reducing serum phosphorus levels for these patients."Real-world survey data collected through the ArdelyxAssist patient services program showed that 63% of patients reported their phosphate levels were better since starting tenapanor. Among patients who reported a change in serum phosphate levels, 69% indicated their outlook on serum phosphate control was better, with improvements attributed to better control, improved bowel movements, and lower pill burden.Source: https://equity-insider.com/2025/03/21/unlocking-the-trillion-dollar-ai-market-what-investors- need-to-know/ CONTACT:
Equity Insider
info @acblanke1DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Avant Technologies Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares Avant Technologies Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Avant Technologies Inc. which were purchased in the open market. MIQ reserves the right to buy and sell, and will buy and sell shares of Avant Technologies Inc. at any time thereafter without any further notice. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material disseminated by MIQ has been approved by the above mentioned company; this is a paid advertisement, and we own shares of the mentioned company that we will sell, and we also reserve the right to buy shares of the company in the open market, or through other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.SOURCES CITED:https://www.mordorintelligence.com/industry-reports/global-regenerative-medicines-market-industryhttps://www.fda.gov/news-events/press-announcements/fda-increases-flexibility-requirements-cell-and-gene-therapies-advance-innovationhttps://www.biospace.com/drug-development/biotech-investors-bet-on-a-2026-rebound-as-deal-activity-accelerateshttps://www.globenewswire.com/news-release/2026/01/20/3221521/0/en/Biotechnology-Market-Size-to-Surpass-USD-9-06-Trillion-by-2035.htmlLogo: https://mma.prnewswire.com/media/2840019/Equity_Insider_Logo.jpg

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Original: Breaking Barriers: How 2026's Top Clinical Leaders Are Disrupting Chronic Disease Markets