Elevidys distribution halted

Elevidys was supposed to be a milestone for Duchenne muscular dystrophy, a fatal muscle-wasting disease that usually begins in childhood. The therapy, made by Sarepta, uses an adeno-associated virus to deliver a shortened dystrophin gene in a single infusion. In June 2024, the FDA expanded its approval to cover Duchenne patients age 4 and older, giving traditional approval for patients who can still walk and accelerated approval for those who cannot. That split now matters more than it first seemed. (fda.gov, cgtlive.com) The trouble became impossible to ignore in 2025. In March, Sarepta disclosed that a young man treated with Elevidys had died after acute liver failure. In June, the company reported a second death, again from acute liver failure, and said both patients had been nonambulatory. Sarepta then stopped shipping Elevidys for nonambulatory patients and paused dosing in its confirmatory ENVISION trial while it worked on a heavier immunosuppression plan meant to blunt liver toxicity. (sarepta.com, cgtlive.com, statnews.com) That still left a narrow story. Two deaths. Both in sicker, older Duchenne patients who could no longer walk. The FDA’s first move fit that story too. On June 25, 2025, the agency said it was investigating the deaths and noted that Elevidys labeling already warned about acute serious liver injury, but not liver failure or death. The question was whether the risk was confined to one subgroup or pointed to something deeper in the platform itself. (fda.gov, cgtlive.com) Then a third death broke that frame. The patient had received SRP-9004, an experimental Sarepta gene therapy for limb-girdle muscular dystrophy, not Elevidys itself. But it used the same AAVrh74 viral shell that carries the genetic payload. By July 18, the FDA said it had three reports of fatal acute liver failure tied to Sarepta AAVrh74 gene therapies and that the deaths appeared to have been caused by the products. The agency put Sarepta’s limb-girdle trials on clinical hold, revoked the company’s platform technology designation, and asked Sarepta to voluntarily stop all Elevidys shipments in the U.S. (fda.gov, fda.gov, biopharmadive.com) Sarepta did not agree at first. On July 19, the company told the Duchenne community it would keep shipping Elevidys for ambulatory patients because it saw no new safety signal in that group. It also argued that the limb-girdle death had been wrongly conflated with Elevidys, since SRP-9004 used a different dose, a different manufacturing process, and targeted a different disease. That was a real distinction, but it was not enough to calm regulators who were now looking at the shared delivery system, not just the brand name on the vial. (sarepta.com, reuters.com) A few days later, Sarepta reversed itself. The company said it would temporarily pause all U.S. Elevidys shipments effective July 22, 2025, so it could answer FDA questions and finish a safety-label supplement. Ten days after that, the FDA partially reopened the door. On July 28, the agency said its investigation had found that the death of an 8-year-old ambulatory boy was unrelated to Elevidys, and it recommended lifting the hold for ambulatory patients while keeping the nonambulatory hold in place. The result was a strange regulatory snapshot: a therapy still available for some children, still blocked for others, and still shadowed by two confirmed Elevidys deaths plus a third fatal liver failure case in a sister program built on the same viral backbone. (cgtlive.com, fda.gov, fda.gov)