Latest news with #AAVrh74
Canada News.Net
a day ago
- Health
- Canada News.Net
FDA urges Elevidys to pause after third death; Sarepta refuses
CAMBRIDGE, Massachusetts: A third patient death linked to gene therapy technology has intensified scrutiny on Sarepta Therapeutics, whose Duchenne muscular dystrophy treatment, Elevidys, remains at the centre of safety concerns. Despite a request from U.S. regulators to halt shipments, the company is refusing to pause deliveries for ambulatory patients. On July 18, the Food and Drug Administration confirmed that it had asked Sarepta to voluntarily stop distributing Elevidys, a gene therapy for Duchenne muscular dystrophy. The request came during a meeting between the agency and the company. Sarepta declined the FDA's request, stating it would continue shipping Elevidys to patients who are able to walk while maintaining an existing pause for non-ambulatory patients — a halt implemented by the company on June 15 after reporting a case of acute liver failure. Sarepta said its decision to continue shipping to ambulatory patients was "based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population." The most recent patient death — a 51-year-old man with limb girdle muscular dystrophy — did not involve Elevidys but another experimental therapy using similar gene technology. The FDA said clinical trials involving limb girdle muscular dystrophy have now been placed on hold due to safety concerns. Earlier this year, two teenage boys receiving Elevidys died of acute liver failure. All three deaths occurred in non-ambulatory patients. These events have raised alarm among regulators, investors, and patient advocacy groups. Elevidys received traditional FDA approval in 2024 for ambulatory patients aged four and older with Duchenne muscular dystrophy. It was also granted accelerated approval for non-ambulatory patients, despite the therapy failing to meet its primary goal in a late-stage trial. In a further regulatory setback, the FDA also revoked Sarepta's platform technology designation for its AAVrh74 gene therapy — a status that would have streamlined approval for similar treatments. Following the news of the third death and the regulatory developments, Sarepta shares plunged 36 percent on July 18 to US$14.08, briefly hitting a more than nine-year low. The FDA is continuing to investigate the risk of acute liver failure linked to Sarepta's platform technology. Some analysts have said this could make patients and families more reluctant to consider the therapy. "Families with Duchenne muscular dystrophy are grappling with a mix of disappointment, concern... and uncertainty about choices they are making for their children or themselves," said Debra Miller, founder of the non-profit CureDuchenne. Sarepta said earlier this week that it is working with the FDA to update Elevidys' packaging to include a warning about liver toxicity risks. However, the company is also facing criticism for its communication handling. During an investor call, analysts questioned why Sarepta had not disclosed the latest patient death earlier in the week when it announced 500 layoffs and financial restructuring. CEO Doug Ingram responded that the death was "neither material nor central" to that announcement. Some analysts, including those at BMO Capital Markets, have warned that how the disclosure was managed could damage Sarepta's credibility with investors.
Newsweek
2 days ago
- Business
- Newsweek
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
Based on facts, either observed and verified firsthand by the reporter, or reported and verified from knowledgeable sources. Newsweek AI is in beta. Translations may contain inaccuracies—please refer to the original content. The U.S. Food and Drug Administration (FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments. The agency announced the action on Friday, citing serious safety concerns and "unreasonable and significant risk" to study participants. The FDA also revoked the company's platform technology designation and requested Sarepta voluntarily halt shipments of its approved drug Elevidys, which the company refused to do. Newsweek reached out to Sarepta Therapeutics via email on Sunday after normal business hours for comment. Why It Matters This regulatory intervention represents a significant safety measure in the gene therapy field, affecting both ongoing clinical trials and an already approved treatment for Duchenne muscular dystrophy. The decision could impact treatment options for families dealing with these devastating muscle-wasting diseases. Duchenne muscular dystrophy is a rare and serious genetic condition that worsens over time, leading to weakness and wasting away of the body's muscles. What To Know The three deaths resulted from acute liver failure in patients treated with either Elevidys or investigational therapies using the same AAVrh74 viral vector technology. One fatality occurred during a clinical trial for limb girdle muscular dystrophy treatment. Elevidys is an adeno-associated virus vector-based gene therapy designed to deliver a gene that leads to production of a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body's muscle cells intact. The drug received traditional approval for ambulatory DMD patients aged 4 and older on June 20, 2024, while maintaining accelerated approval for non-ambulatory patients since June 22, 2023. Under accelerated approval, companies must conduct confirmatory studies to verify clinical benefit. Given the new safety concerns, the FDA has notified Sarepta that the drug's indication should be restricted to ambulatory patients only. The FDA revoked the platform technology designation for Sarepta's AAVrh74 Platform Technology because the preliminary evidence is insufficient to demonstrate that the technology has the potential to be incorporated in multiple drugs without adverse safety effects. What People Are Saying FDA Commissioner Marty Makary, M.D., M.P.H.: "Today, we've shown that this FDA takes swift action when patient safety is at risk. We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges." Dr. Vinay Prasad, Director of FDA's Center for Biologics Evaluation and Research: "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." FILE - The Food and Drug Administration seal is seen at the Hubert Humphrey Building Auditorium in Washington, Tuesday, April 22, 2025. FILE - The Food and Drug Administration seal is seen at the Hubert Humphrey Building Auditorium in Washington, Tuesday, April 22, 2025. AP Photo/Jose Luis Magana, File What Happens Next The FDA will continue investigating the risk of acute liver failure and other serious outcomes following gene therapies using Sarepta's AAVrh74 platform technology. The agency has committed to taking additional regulatory actions as needed to protect patients. For current Elevidys patients, the FDA is focusing its safety investigation on ambulatory patients while restricting the drug's use in non-ambulatory patients.
Business Insider
4 days ago
- Health
- Business Insider
FDA investigating risk of acute liver failure with Sarepta gene therapies
As of July 18, 2025, the Food and Drug Administration said it has received three reports of fatal acute liver failure following treatment of patients with Sarepta (SRPT) AAVrh74 gene therapies that appear to have been caused by the gene therapy products as a result of acute liver failure. The agency added, 'FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following gene therapies using Sarepta's AAVrh74 gene therapy products, and the need for further regulatory action(s).' Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week.
Business Wire
04-06-2025
- Business
- Business Wire
U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta's Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & Drug Administration. 'This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,' said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. 'The designation underscores and reinforces the consistency of the data we have seen with this AAVrh74 in multiple clinical programs and is yet another example of Sarepta's continued commitment to accelerating the development of potentially transformative treatments for patients with rare genetic diseases like LGMD type 2E/R4.' In keeping with FDA's pursuit of science-driven efficiency, the platform technology designation program is a critical tool that will help support rare disease therapies by streamlining drug development, manufacturing, and review processes for drug product applications by incorporating data from designated technologies. To be eligible for this designation, the technology must be a well-understood and reproducible technology – such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies – and can support the development of multiple drugs or biologics. A platform is also eligible for consideration if it is incorporated in an approved drug and preliminary evidence demonstrates that the technology has the potential to be incorporated in or utilized by more than one drug without an adverse effect on quality, manufacturing, or safety, including using the same manufacturing process. Once granted, the designation enables sponsors to leverage prior data from the platform to support investigational new drug applications (INDs), new drug applications (NDAs) or biologic license applications (BLAs). About SRP-9003 (bidridistrogene xeboparvovec) SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications. 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 leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) 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. 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. 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, product candidates, the potential benefits of AAVrh74 and the potential for our therapies to be transformative for patients living with rare diseases. 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: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; 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; 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 regulatory decisions by, and any halts imposed by, 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.
