The US Food and Drug Administration (FDA) is investigating the death of an 8-year-old boy who received Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy. The patient died on June 7 in Brazil, but the agency has concluded that his death is unrelated to treatment with the therapy.
Sarepta partners with Roche outside of the US to market its treatments. Roche reported that a medical professional who treated the boy had assessed the death as unrelated to Elevidys treatment. The company has also reported two other fatalities linked to the gene therapy, but these were non-ambulatory teenage boys in the US.
Following these incidents, Sarepta announced it will pause shipments of its experimental gene therapy SRP-9004 and comply with an FDA request to halt all shipments of Elevidys in the US. Roche has also paused shipments outside of the US.
The FDA’s investigation into the boy’s death is ongoing, but the agency has not commented further on the incident.
Source: https://www.reuters.com/business/healthcare-pharmaceuticals/fda-probes-death-patient-sareptas-elevidys-partner-roche-says-death-unrelated-2025-07-25