Second Patient Dies After Receiving Sarepta’s Elevidys Gene Therapy for DMD

A second patient has died after receiving Sarepta Therapeutics’ gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD). Both deaths occurred in non-ambulatory (wheelchair-bound) patients and were caused by acute liver failure, a known side effect of adeno-associated virus (AAV)-based gene therapies like Elevidys124.

Following the second death, Sarepta has suspended Elevidys use in non-ambulatory patients in the U.S. and halted dosing in the Phase III ENVISION trial, which is required for the therapy's confirmatory FDA approval in this patient group14.

Roche, Sarepta's ex-U.S. partner, has discontinued Elevidys for commercial use in non-ambulatory patients outside the U.S1. European regulators had previously put some Elevidys trials on temporary holds after the first reported death1.

The most recent patient was a 15-year-old in the ENVISION trial. Sarepta aims to amend the trial protocol to include prophylactic use of the immunosuppressant sirolimus to help manage the risk of liver toxicity12.

Both patient deaths followed a previously unreported severity of acute liver injury associated with Elevidys. The incident has caused Sarepta’s stock to drop significantly and has left the DMD patient community concerned and divided regarding the therapy’s future23.

Sources:

1. https://www.fiercepharma.com/pharma/sarepta-records-second-death-after-dmd-gene-therapy-elevidys-stops-dosing-half-patients

2. https://www.biospace.com/drug-development/second-patient-dies-after-receiving-sareptas-dmd-gene-therapy-elevidys

3. https://www.statnews.com/2025/06/16/duchenne-muscular-dystrophy-community-reacts-second-death-sarepta-therapeutics-elevidys/

4. https://www.statnews.com/2025/06/15/duchenne-sarepta-gene-therapy-elevidys-patient-death/