FDA Investigates Elevidys Death; Sarepta and Roche Deny Link to Gene Therapy

The FDA is investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics' Elevidys, a gene therapy for Duchenne muscular dystrophy1 3 5.

The death occurred on June 7, 2025, and upon FDA request, Sarepta and Roche suspended Elevidys shipments in the U.S. and Brazil while safety concerns are assessed1 3.

Both Sarepta and Roche have publicly stated—citing the treating physician and Brazilian authorities—that the death was unrelated to Elevidys, instead attributing it to severe infection (likely influenza A) exacerbated by immunosuppression1 5.

The boy was not part of any Elevidys clinical trials; the case was reported through official regulatory channels to both FDA and Brazilian health authorities1 5.

This is the third reported patient death this year related to Sarepta's gene therapy programs; the previous death was linked to a different muscular dystrophy treatment using the same virus vector platform2 4.

Debate exists as the FDA investigates, with market withdrawal being considered, and the gene therapy’s approval had been controversial due to disputed safety and efficacy concerns2 4.