Sarepta Therapeutics will voluntarily and temporarily pause all shipments of Elevidys gene therapy for Duchenne muscular dystrophy in the U.S., following a public dispute with the FDA.
The FDA issued a press release on Friday afternoon stating that agency leadership had met with Sarepta and requested it voluntarily stop all shipments of Elevidys that day. According to the FDA, the company refused to do so.
Sarepta followed up with its own press release on Friday, confirming that the company had received the informal request from the agency to halt Elevidys shipments but that because the data shows no new or changed safety signals in the ambulant patient population, the company will continue to ship Elevidys to the ambulant population.
According to Sarepta, the company had already made a “conservative decision” — announced last month — to pause shipments of Elevidys for non-ambulant patients while working with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure. This decision had been made following the second non-ambulatory patient death following treatment with Elevidys.
In the FDA’s Friday press release, the agency also announced that it had placed Sarepta’s investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold following a recently revealed patient death in phase 1 study of SRP-9004. The FDA also revoked Sarepta’s platform technology designation which had been awarded to the company in June for the rAAVrh74 viral vector used in SRP-9003 — the only limb-girdle muscular dystrophy program that had survived Sarepta’s recent pipeline cuts.
But by Monday, the story had changed. Sarepta issued a press release stating that, effective close of business Tuesday, July 22, 2025, the company will pause all shipments of Elevidys to respond to any requests for information and allow Sarepta and the FDA to complete the Elevidys safety labeling supplement process.
“It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process,” said Doug Ingram, Sarepta CEO.
The company said it will provide timely updates as additional information becomes available.
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