Latest news with #AAVrh74PlatformTechnology
Yahoo
21-07-2025
- 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typically granted to technologies with broad therapeutic promise that can streamline regulatory review, was rescinded because, given the new safety information, the preliminary evidence was deemed insufficient to demonstrate that the AAVrh74 Platform Technology could be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.
Newsweek
21-07-2025
- 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.
