The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns.
The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy. – Read More –
Roche has not yet provided detailed information
Sarepta felt the need to make a statement on the issue on July 25, 2025.
The death of this patient was deemed unrelated to treatment with ELEVIDYS. As reported yesterday by Naomi Kresge at Bloomberg News:
Roche Holding AG says the recent death of a patient in Brazil who had been treated with gene therapy Elevidys for Duchenne muscular dystrophy is unrelated to the treatment. – Statement on ELEVIDYS –
- The boy wasn’t a clinical trial participant; reporting physician assessed his death as being unrelated to the gene therapy, Roche says in statement
- Death was reported to health authorities
- Roche, which markets Sarepta’s Duchenne treatment Elevidys outside the US, declines to comment on the boy’s age or details of the case
Sarepta reported this event to FDA on June 18, 2025, via the FDA’s postmarketing electronic database, FAERS.
