Latest news with #LGMD
Yahoo
3 days 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.
Yahoo
3 days ago
- Business
- Yahoo
Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys
The ground shook for Sarepta Therapeutics, Inc. (NASDAQ:SRPT) last week as the U.S. Food and Drug Administration (FDA), alarmed by a third patient death tied to its gene therapy platform, pulled a rare move: requesting a voluntary halt to Elevidys shipments. The company's response? A resounding 'no,' igniting a major regulatory clash with severe implications for patients and investors alike. The upheaval began on Friday when the U.S. Food and Drug Administration (FDA) made a rare and impactful announcement: it had placed Sarepta's investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD) on a clinical hold. This decision stemmed from serious safety concerns, including three patient deaths potentially linked to these products, indicating that study participants faced or would be exposed to an unreasonable and significant risk of illness or injury. In light of these developments, the FDA also formally requested Sarepta to voluntarily halt all shipments of Elevidys, the company's approved gene therapy. However, in a move that set the stage for a major regulatory clash with severe implications for both patients and investors, Sarepta definitively refused this three fatalities, which prompted the FDA's strong action, have resulted from acute liver failure in individuals who had received either Elevidys or an investigational gene therapy utilizing the same AAVrh74 serotype, a viral vector used in gene delivery. Notably, one of these deaths occurred during a clinical trial for LGMD, conducted under an investigational new drug application. Adding to Sarepta's challenges, the FDA further revoked the platform technology designation for the company's AAVrh74 Platform Technology. This designation, typically granted to technologies with broad therapeutic promise that can streamline regulatory review, was rescinded because, given the new safety information, the preliminary evidence was deemed insufficient to demonstrate that the AAVrh74 Platform Technology could be incorporated into or utilized by multiple drugs without adverse effects on safety. Elevidys had received traditional approval in June 2024 for ambulatory Duchenne muscular dystrophy (DMD) patients aged four years and older with a confirmed DMD gene mutation. Previously, in June 2023, it secured accelerated, conditional approval for non-ambulatory DMD patients. However, continued approval for non-ambulatory patients is contingent upon verification of clinical benefit through confirmatory trials. With the emergence of this new safety data, the FDA notified Sarepta that Elevidys's indication should now be restricted solely to ambulatory patients. The FDA is continuing its investigation into the risk of acute liver failure, including severe outcomes such as hospitalization and death, associated with gene therapies employing Sarepta's AAVrh74 Platform Technology, and is prepared to take further regulatory actions as necessary. Despite the FDA's stance, Sarepta issued a statement on Friday affirming its decision to continue shipping Elevidys to the ambulatory patient population. The company asserted that its 'comprehensive scientific interpretation of the data... shows no new or changed safety signals in the ambulant patient population.' Sarepta also expressed its commitment to ongoing discussions and information sharing with the FDA to advance their 'shared purpose of protecting patient safety and informed access to care.' The company clarified that the most recent fatal event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004, which is designed to treat a different condition, LGMD Type 2D. Sarepta emphasized that SRP-9004 is administered at a different dose and manufactured using a distinct process from Elevidys. Furthermore, the LGMD study participant who passed away was not treated with Elevidys, and dosing for the SRP-9004 trial had already concluded at the time of death. Sarepta reported this acute liver failure (ALF) event as a life-threatening case to the FDA on June 20 and subsequently notified the agency of the death on July 3. Looking ahead, Sarepta plans to submit the findings of an expert panel and a proposed protocol to the FDA. They will also discuss a proposal to collect additional data on a modified treatment regimen in a new cohort (Cohort 8) of the ENDEAVOR study (Study SRP-9001-103). This is intended as a pathway to re-establish dosing for non-ambulant patients in the future. Price Action: As of Monday's premarket session, Sarepta's stock was down 6.79% at $13.12, reflecting the ongoing concerns and market reaction to these significant developments. Read Next:Photo via Shutterstock Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? SAREPTA THERAPEUTICS (SRPT): Free Stock Analysis Report This article Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
Business Insider
5 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
6 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
6 days ago
- Business
- Yahoo
SRPT Stock Soars on Unveiling New Restructuring Plan, Pipeline Pivots
Shares of Sarepta Therapeutics SRPT surged more than 30% in after-market trading yesterday after the company announced a strategic restructuring plan focused on pipeline reprioritization and supporting long-term financial sustainability. SRPT to Cut Costs & Focus on siRNA Pipeline Sarepta's restructuring plan aims to save about $400 million annually starting in 2026. To achieve this target, the company has decided to lay off 36% of its workforce — around 500 employees — which is expected to save nearly $120 million per year. SRPT plans to generate around $300 million in annual savings by reprioritizing its pipeline. Concerning the pipeline, Sarepta has decided to pause development of most of its gene-therapy candidates for limb-girdle muscular dystrophy (LGMD). The company, however, intends to move forward with SRP-9003, its gene therapy candidate for patients with LGMD type 2E/R4 (LGMD2E/R4, or beta sarcoglycanopathy). A regulatory filing with the FDA remains on track for this therapy before year-end. SRPT has now shifted focus to its siRNA programs, which were acquired as part of a multi-billion-dollar licensing deal with Arrowhead Pharmaceuticals ARWR. After closing the Arrowhead deal in February, Sarepta acquired exclusive rights to four clinical-stage siRNA programs, each being evaluated in separate phase I/II studies. These include SRP-1001 in facioscapulohumeral muscular dystrophy (FSHD), SRP-1002 in idiopathic pulmonary fibrosis (IPF), SRP-1003 in myotonic dystrophy type 1 (DM1) and SRP-1004 in spinocerebellar ataxia 2 (SCA2). Initial data from studies on SRP-1001 and SRP-1003 are expected before year-end. Sarepta also acquired three preclinical programs from Arrowhead, including SRP-1005 (for Huntington's disease), which is set to enter clinical development early next year. While Sarepta expects to incur severance and one-time charges of $32 million to $37 million, it still anticipates saving over $100 million in annual costs this year. For full-year 2026, Sarepta expects the combined adjusted R&D and SG&A expenses to be in the range of $800-$900 million. SRPT Stock Performance Sarepta's motivation behind these moves is clear — this company-wide reorganization aims to address the setback stemming from safety concerns around Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). The latest surge in stock price reflects investor optimism regarding the restructuring plan's ability to stabilize the company's financial profile and growth trajectory. Year to date, Sarepta's shares have plunged 85% compared with the industry's 2% decline. Image Source: Zacks Investment Research Sarepta to Update Elevidys Label With Black Box Warning Investor sentiment toward the stock has worsened significantly after two patient deaths were linked to Elevidys, both caused by acute liver failure (ALF) in non-ambulatory DMD patients. While Sarepta has already suspended both clinical and commercial Elevidys dosing for non-ambulatory patients, the therapy has been mandated by the FDA to carry a black box warning for ALF and acute liver injury. To address this safety issue, Sarepta is working to create a new protocol with an enhanced immunosuppression regimen to make Elevidys administration safer for non-ambulatory DMD patients. The company plans to submit these findings to the FDA in hopes of resuming dosing in the non-ambulation population. Sarepta developed Elevidys in partnership with pharma giant Roche RHHBY. In 2019, SRPT and Roche entered into a licensing agreement to develop Elevidys. Per the agreement, Roche has exclusive rights to launch and market the therapy in non-U.S. markets. Sarepta Provides Q2 Preliminary Numbers Alongside the restructuring plan, Sarepta also issued preliminary/unaudited figures for the second quarter of 2025. The company reported total net product revenues of $513 million, which beat both the Zacks Consensus Estimate of $506 million and our model estimate of $507 million. The unaudited revenue numbers include $282 million from Elevidys. The Zacks Consensus Estimate and our model estimate for Elevidys' sales are pegged at $274 million and $280 million, respectively. Sarepta also reported preliminary combined adjusted R&D and SG&A expenses of around $294 million for the quarter. As of the end of June 2025, unaudited cash and short-term investments totaled approximately $850 million. The company plans to report full second-quarter results early next month. Sarepta Therapeutics, Inc. Price Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote SRPT's Zacks Rank Sarepta currently carries a Zacks Rank #4 (Sell). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Roche Holding AG (RHHBY) : Free Stock Analysis Report Sarepta Therapeutics, Inc. (SRPT) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research
