The FDA is investigating the death of an 8-year-old boy who received Elevidys, a gene therapy developed by Sarepta Therapeutics to treat Duchenne muscular dystrophy.
The child died June 7, prompting the FDA to request a voluntary suspension of the therapy while it investigates potential safety concerns, according to a July 25 news release from the agency.
The distribution pause comes in the wake of ongoing discussions between Sarepta and the FDA around safety labeling. On July 21, Sarepta halted shipments of Elevidys, allowing time to respond to the FDA’s inquiries and complete a labeling safety supplement process.
The move came after multiple reports of patient deaths, including two earlier in 2025 tied directly to Elevidys and a third death involving a separate trial for another Sarepta gene therapy.
