Sarepta Disregards FDA Request to Stop Shipping Elevidys
Sarepta is brushing off a 7/18 “informal” request from FDA to voluntarily halt shipment of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy. The request was in response to two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male Duchenne patients with the gene therapy (see earlier story)..
Sarepta hinted that it was caught off guard by FDA’s request, indicating that it first heard of the potential request “earlier in the day at the same time the public and our patient communities did, through media reports.” Noting that it had already “paused 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.”
The company was under FDA’s microscope after recently reporting a third gene therapy patient death, which it says was related to a different therapy called SRP-9004, a clinical-stage therapy that is intended to treat a different disease (Limb-girdle muscular dystrophy Type 2D). In distinguishing the product, the company says SRP-9004 is administered using a different dose, and is manufactured using a different process. “Additionally, in a timely manner, Sarepta reported this ALF [acute liver failure] 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,” it says.
The regulatory ball is in FDA’s court on whether the agency will proceed to escalate the matter and take regulatory action to remove Elevidys from the market altogether. On 7/18, an HHS official told FDA Webview that the agency was taking a “hard look” at pulling the gene therapy from the market.
Meanwhile, FDA has placed Sarepta’s investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold, and it revoked its platform technology designation. “The three deaths appear to have been a result of acute liver failure in individuals treated with Elevidys or investigational gene therapy using the same AAVrh74 serotype that is used in Elevidys,” an FDA notice says. “Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits.”