FDA Investigates Patient Deaths Linked to Sarepta's Elevidys Gene Therapy for Duchenne Muscular Dystrophy

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The FDA is investigating two reported deaths of non-ambulatory pediatric male patients with Duchenne muscular dystrophy (DMD) who developed fatal acute liver failure after receiving Sarepta Therapeutics’ gene therapy, Elevidys123.

Both fatalities occurred within two months of treatment, with patients presenting elevated transaminases and requiring hospitalization13.

The deaths were reported in March and June 2025, involving teenage boys who were non-ambulatory (unable to walk independently) due to disease progression23.

Liver toxicity is a known risk with adeno-associated virus vector-based gene therapies such as Elevidys, but the current U.S. prescribing information only warns of serious liver injury—not specifically liver failure or death12.

Following these deaths, Sarepta temporarily halted shipments of Elevidys for non-ambulatory patients and paused dosing in its phase 3 ENVISION confirmatory trial4.

Sarepta is updating the prescribing information to include the recent deaths and is working to develop an enhanced immunosuppressive regimen to mitigate risk for non-ambulatory patients4.

The FDA is evaluating the need for further regulatory action, potentially including stronger warnings (such as a black box warning), but no specific measures have yet been announced23.

Sources:

1. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-non-ambulatory-duchenne-muscular-dystrophy-patients

2. https://www.fiercepharma.com/pharma/fda-investigates-deaths-after-sarepta-duchenne-gene-therapy-elevidys

3. https://www.biospace.com/fda/fda-reviews-sareptas-elevidys-after-second-patient-death

4. https://www.cgtlive.com/view/fda-announces-investigation-deaths-following-treatment-sarepta-dmd-gene-therapy-elevidys