Elevidys Liver Failure Risks Added to FDA Black Box Warning

Federal regulators have also asked manufacturer to stop shipping the muscular dystrophy drug, following three deaths.

Sarepta Therapeutics has agreed to stop shipping its Duchenne muscular dystrophy (DMD) treatment Elevidys, and will add an FDA black box warning about the risk of severe liver injury risks.

Elevidys (delandistrogene moxeparvovec-rokl) was developed by Sarepta Therapeutics as a one-time gene therapy approved to treat DMD in patients ages 4 and older. Duchenne muscular dystrophy is a rare genetic condition that leads to progressive muscle weakening due to a lack of dystrophin, a protein essential for muscle function.

After the FDA granted accelerated approval in June 2023, Elevidys became the first gene therapy approved for Duchenne muscular dystrophy. The decision followed an internal dispute, with the head of the FDA’s Center for Biologics Evaluation and Research overruling staff reviewers who questioned whether the treatment was ready. In 2024, the FDA expanded its approval to cover most U.S. patients with DMD.

However, earlier this year, Sarepta Therapeutics acknowledged that a 16-year-old patient treated with Elevidys had died from acute liver failure, in a press release issued March 18, 2025. In the following months, at least twomore patients died under similar circumstances.

On July 16, Sarepta issued another press release stating that it had halted Elevidys shipments for non-ambulatory DMD patients at the FDA’s request, and wlll add a black box warning about the risk of severe liver injury.

However, the FDA issued a news release two days later, stating that it had placed Elevidys clinical trials on hold, and requested that Sarepta voluntarily stop all Elevidys shipments to both non-ambulatory and ambulatory patients immediately.

Despite initially refusing to halt shipments to ambulatory patients, Sarepta eventually agreed to pause all Elevidys shipments, effective July 22.

“Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.”

-Vinay Prasad, M.D., M.P.H., Director of the FDA’s Center for Biologics Evaluation and Research

More than 800 patients have received Elevidys in either clinical trials or as a prescribed therapy. The possibility of acute liver failure leading to death as a side effect had never been reported prior to the first patient death announced on March 18.

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