FDA Approves New Safety Warning and Revised Indication for Sarepta’s DMD Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has approved new labeling for Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, limiting its use to ambulatory patients 4 years of age and older.

The revised labeling also adds a boxed warning describing the risk of serious liver injury and acute liver failure, including fatal outcomes.

The FDA’s actions follow reports of fatal acute liver failure in non-ambulatory patients treated with the gene therapy. In June 2025, the agency issued a safety communication after two reports of fatal acute liver failure in non-ambulatory pediatric males with Duchenne muscular dystrophy who had received Elevidys. In response, Sarepta voluntarily paused distribution of the therapy for non-ambulatory patients.

In both fatal cases, patients developed markedly elevated liver enzymes and required hospitalization within two months of Elevidys infusion. Another serious, nonfatal case of acute liver injury involved complications such as mesenteric vein thrombosis, bowel ischemia and necrosis, and portal hypertension.

The revised labeling includes new safety information and monitoring recommendations, including weekly liver function tests for at least three months after treatment. It also advises that patients remain near an appropriate medical facility for at least two months post-infusion. Additionally, the labeling recommends weekly testing for cardiac injury during the first month after treatment.

The FDA said Elevidys should not be used in patients with deletions involving DMD exons 8 and/or 9. It also is not recommended for patients with preexisting liver impairment, recent vaccinations, or recent or active infections.

The agency is requiring Sarepta to conduct a postmarketing observational study to assess the risk of serious liver injury further. The study will enroll about 200 patients with Duchenne muscular dystrophy and follow them for at least 12 months after receiving Elevidys, with periodic liver function assessments.