Latest news with #SRP-9004
Yahoo
a day ago
- Health
- Yahoo
Sarepta refuses to pull gene therapy despite FDA order
Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the US Food and Drug Administration (FDA). Speaking on 18 July, shortly after the FDA ordered the pull for Elevidys, Sarepta said it will continue to ship Elevidys to the ambulant population as studies show no signs of new or changed safety signals, adding it first heard of this potential request earlier in the day through media reports. A statement from the company reads: '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. "This guides every decision we make, as evidenced by our conservative decision to pause shipments of Elevidys for non-ambulant patients while we work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure (ALF).' Trial of LGMD therapy also put on hold The FDA has also put a hold on Sarepta's gene therapy SRP-9004 in limb girdle muscular dystrophy (LGMD) after a patient died in an early-stage study due to ALF. Speaking about the patient death in the Phase I trial of SRP-9004, the company's statement added: 'We recognise that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant LGMD patient. "We also want to clarify that this tragic event occurred in a Phase I clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical-stage therapy that is intended to treat a different disease, is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with Elevidys, and the dosing for the SRP-9004 trial had concluded at the time of his death.' This patient's death, which was reported to the FDA on 3 July, is the third that has impacted Sarepta's gene therapy programme, with the first two deaths earlier this year in patients treated with Elevidys, both also due to ALF. Both Elevidys and SRP-9004 are adeno-associated virus (AAV) gene therapies that use the same AAVrh74 serotype. The FDA's Center for Biologics Evaluation and Research (CBER) director Dr Vinay Prasad, said: 'Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.' This comes just days after Sarepta said it has agreed to change the black box on the Elevidys label to include ALF and acute liver injury (ALI) warnings. In the same announcement, Sarepta said it would be cutting 500 jobs as part of company restructuring. Speaking after the third patient death was announced, GlobalData healthcare analyst Momna Ali said the recent setbacks for Sarepta put the cell and gene therapy (CGT) sector at a 'crossroads'. Ali said: 'Following Sarepta's announcement of a third patient death, a new label for Elevidys and them laying off 500 employees, or 36% of its workforce, shelving parts of its pipeline to save $420m, and shifting focus from gene therapy to siRNA programmes, this is going to put the CGT sector at a pivotal crossroad. 'While the scientific potential of CGT therapies remains extraordinary, this moment serves as a reminder of the complexity, cost, and responsibility involved. There is a lot of buzz around therapies 'beyond the pill'; however, for the landscape to keep evolving at the pace it has been in the last 3-5 years, there has to be greater transparency, patient safety, and sustainable innovation – otherwise, it'll be met with more setbacks.' The news of the LGMD death came to light on 17 July in media reports. After this was reported, Sarepta's stock has dropped 43.21%, from $22.54 at market open on 17 July to $12.80 at market open on 21 July. The FDA also said it has revoked the platform technology designation for Sarepta's AAVrh74 Platform Technology. "Sarepta refuses to pull gene therapy despite FDA order" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Business Insider
4 days ago
- Business
- Business Insider
Sarepta says will continue to ship Elevidys to ambulant population
Sarepta (SRPT) Therapeutics issued a statement which reads in part: 'Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration to voluntarily halt shipment of Elevidys, our gene therapy for Duchenne muscular dystrophy, in the U.S. We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports…Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care. We recognize that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant Limb-Girdle Muscular Dystrophy (LGMD) patient. We also want to clarify that this tragic event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical stage therapy that is intended to treat a different disease (LGMD Type 2D), is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with ELEVIDYS, and the dosing for the SRP-9004 trial had concluded at the time of his death.' Elevate Your Investing Strategy:
Business Wire
4 days ago
- Health
- Business Wire
Sarepta Therapeutics Provides Statement on ELEVIDYS
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment of ELEVIDYS (delandistrogene moxeparvovec), our gene therapy for Duchenne muscular dystrophy (Duchenne), in the U.S. We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports. 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. This guides every decision we make, as evidenced by our conservative decision to pause shipments of ELEVIDYS for non-ambulant patients while we work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure. Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship ELEVIDYS to the ambulant population. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care. We recognize that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant Limb-Girdle Muscular Dystrophy (LGMD) patient. We also want to clarify that this tragic event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical stage therapy that is intended to treat a different disease (LGMD Type 2D), is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with ELEVIDYS, and the dosing for the SRP-9004 trial had concluded at the time of his death. Additionally, in a timely manner, Sarepta reported this ALF event as a life-threatening case to FDA on June 20, 2025, and further followed up with notification to FDA of the death on July 3, 2025, in accordance with applicable law and our commitment to full regulatory transparency. ELEVIDYS is the only approved gene therapy for individuals devastated by Duchenne, a rare, progressive and ultimately fatal disease. We are 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 Therapeutics Sarepta 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 Statements This 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, ELEVIDYS, and expected plans, including our plan to continue to ship ELEVIDYS to the ambulant population and continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care. 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; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; we may not be able to comply with all FDA requests in a timely manner or at all; 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 Information We 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.
Yahoo
4 days ago
- Business
- Yahoo
Sarepta share price drops again after third gene therapy death
Sarepta Therapeutics' share price has fallen again following the death of a third patient dosed with its gene therapy treatment. A 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 died after receiving Sarepta's gene therapy candidate SRP-9004 in a Phase I trial, according to Bloomberg. Sarepta Therapeutics was approached for comment by Clinical Trials Arena but did not respond before publication. This comes after two deaths were announced following treatment with Sarepta's Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec). Both patient deaths, the first announced in March 2025 and the second in June 2025, were due to acute liver failure (ALF). Elevidys and SRP-9004 both use the same recombinant adeno-associated viral vector (AAV) in AAVrh74. Reports state that Sarepta has informed regulators and clinical investigators of the latest death after dosing with SRP-9004. Earlier this week, Sarepta said it was cutting 500 jobs as part of its restructuring and pipeline prioritisation plan, as well as updating the label for Elevidys. The company is engaging with the US Food and Drug Administration (FDA) regarding the label update for the gene therapy, which includes adding a black box warning for acute liver injury (ALI) and ALF. Nasdaq-listed Sarepta saw its share price jump 19.53%, rising from $18.38 at the close of 16 July to $21.97 on July 17, following news of the restructuring and new Elevidys label. However, shares plunged 22.62%, to open at $17.00 on 18 July after the company announced the death of the patient in the clinical trial. In May 2025, the FDA announced that American haematologist oncologist Vinay Prasad will lead its Center for Biologics Evaluation and Research (CBER), the division responsible for regulating gene therapies and vaccines. Prasad has openly criticised Elevidys, primarily about the clinical evidence and the FDA's decision to approve the therapy. CGT sector at a 'crossroads' GlobalData healthcare analyst Momna Ali said the recent setbacks for Sarepta put the cell and gene therapy (CGT) sector at a 'crossroads'. Ali said: 'Following Sarepta's announcement of a third patient death, a new label for Elevidys and them laying off 500 employees, or 36% of its workforce, shelving parts of its pipeline to save $420m, and shifting focus from gene therapy to siRNA programmes, this is going to put the CGT sector at a pivotal crossroad. "While the scientific potential of CGT therapies remains extraordinary, this moment serves as a reminder of the complexity, cost, and responsibility involved. There is a lot of buzz around therapies 'beyond the pill'; however, for the landscape to keep evolving at the pace it has been in the last 3-5 years, there has to be greater transparency, patient safety, and sustainable innovation - otherwise, it'll be met with more setbacks.' Pfizer pulled an investigational DMD therapy last year after the death of a patient in a Phase II trial and a Phase III trial failed to meet its primary endpoint. In May 2025, a patient in Rocket Pharmaceuticals' pivotal Phase II trial of gene therapy for Danon disease died after suffering a serious adverse event (AE). "Sarepta share price drops again after third gene therapy death" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
