Due to two documented incidents of severe liver failure that resulted in death, Sarepta Therapeutics has stopped shipping its gene treatment ELEVIDYS for individuals with non-ambulatory Duchenne muscular dystrophy.
The patient who died most recently was 15 years old. In order to allow for protocol changes and regulatory considerations, Sarepta has decided to halt the ENVISION Phase 3 confirmatory trial (SRP-9001-303), which involves adult ambulatory and non-ambulatory adults, due to these serious adverse events. – Read More: Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe –
The two fatal cases were reported in June 2025, with the FDA issuing a safety communication on June 24, 2025, to investigate the potential risk of acute liver failure associated with ELEVIDYS. In response, Sarepta paused its ENVISION Phase 3 trial to update protocols and consult with regulators. The company has also temporarily suspended distribution of ELEVIDYS for non-ambulatory patients until a new immunosuppressive regimen can be approved.
Learn More: After Second Patient Death, Duchenne Muscular Dystrophy Communities Want Answers About Elevidys Gene Therapy
