Sarepta clarifies that latest patient death was unrelated to Elevidys

Following the U.S. FDA’s announcement that it was investigating the death of a boy with Duchenne muscular dystrophy who had received Elevidys, Sarepta has stepped in to clarify that the death was not related to treatment with the gene therapy.

On Friday evening, the FDA issued a brief press release stating that it was investigating the June 7, 2025 death of an 8-year-old boy who received Elevidys.

Sarepta then issued its own press release, citing a Reuters report in which Roche, which owns the ex-U.S. rights to Elevidys, confirmed that the Brazilian patient was not participating in a clinical trial and that the reporting physician had assessed the death as unrelated to treatment with the gene therapy. Sarepta said it reported this event to FDA on June 18, 2025, via the FDA’s postmarketing electronic database, FAERS.

Last week, Sarepta announced it would voluntarily and temporarily pause all shipments of Elevidys in the U.S., following a public dispute with the FDA. The company has reported three patient deaths due to acute liver toxicity — two cases that occurred in non-ambulatory Duchenne patients who had received Elevidys and a third 51-year-old patient with limb-girdle muscular dystrophy who was participating in a clinical trial of SRP-9004.