Latest news with #Duchenne muscular dystrophy
Yahoo
2 days ago
- Business
- Yahoo
Sarepta will resume gene therapy shipments after FDA review of recent patient death
WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
The Independent
2 days ago
- Business
- The Independent
Sarepta will resume gene therapy shipments after FDA review of recent patient death
Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Associated Press
2 days ago
- Business
- Associated Press
Sarepta will resume gene therapy shipments after FDA review of recent patient death
WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Yahoo
5 days ago
- Health
- Yahoo
Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS
CAMBRIDGE, Mass., July 25, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: Just before 6:00 p.m. ET today, the U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy. The death of this patient was deemed unrelated to treatment with ELEVIDYS. As reported yesterday by Naomi Kresge at Bloomberg News: Roche Holding AG says the recent death of a patient in Brazil who had been treated with gene therapy Elevidys for Duchenne muscular dystrophy is unrelated to the treatment. * The boy wasn't a clinical trial participant; reporting physician assessed his death as being unrelated to the gene therapy, Roche says in statement* Death was reported to health authorities* Roche, which markets Sarepta's Duchenne treatment Elevidys outside the US, declines to comment on the boy's age or details of the case Sarepta reported this event to FDA on June 18, 2025, via the FDA's postmarketing electronic database, FAERS. At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies, and do so in accordance with applicable law and commitment to full regulatory transparency. ELEVIDYS is the only approved gene therapy for families and children devastated by Duchenne, a rare, progressive and ultimately fatal disease. We remain committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy. About ELEVIDYS (delandistrogene moxeparvovec-rokl)ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS:Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Forward-Looking StatementsThis statement contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials and ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading "Risk Factors" in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Source: Sarepta Therapeutics, Inc. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
5 days ago
- Business
- Yahoo
Sarepta stock falls after Elevidys fails to get backing by EU regulators
-- Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock declined 13% Friday after European regulators recommended against approving Elevidys, the company's gene therapy for Duchenne muscular dystrophy. The European Medicines Agency's drug advisory committee (CHMP) rejected Elevidys for children aged 3 to 7 years who are able to walk, the agency announced Friday. The therapy, which is marketed outside the U.S. by Roche Holding AG (SIX:ROG), was seeking approval for treating the rare genetic disease that causes progressive muscle weakness. This setback follows closely on the heels of the U.S. Food and Drug Administration's recent request for a pause on Elevidys use in the United States. Sarepta had already announced a temporary halt on U.S. shipments earlier this week. Roche has also paused shipments in jurisdictions that reference FDA approvals, further limiting the therapy's market reach. The Swiss pharmaceutical giant had been responsible for commercializing the treatment in markets outside the U.S. after partnering with Sarepta. Duchenne muscular dystrophy is a rare genetic disorder that primarily affects boys and leads to progressive muscle deterioration. The condition is ultimately fatal, with patients typically experiencing serious mobility limitations and shortened lifespans. The dual regulatory setbacks in both the U.S. and European markets represent significant challenges for Sarepta's commercialization strategy for Elevidys, which had been positioned as a breakthrough treatment option for the debilitating condition. Related articles Sarepta stock falls after Elevidys fails to get backing by EU regulators Victoria's Secret Exposed: The Warning Sign Behind the Stock's 52% Collapse Surge of 50% since our AI selection, this chip giant still has great potential Sign in to access your portfolio
