Sarepta Plunges 43.26% After Second Liver Failure Death

On June 16, 2025, Sarepta's stock experienced a significant drop of 43.26% in pre-market trading.

Sarepta Therapeutics reported a second case of liver failure death following its gene therapy treatment for Duchenne muscular dystrophy. This incident prompted the company to pause its clinical trial for the treatment, which is known as Elevidys. The pause comes after two reported cases of fatal liver failure, both occurring in non-ambulatory individuals with Duchenne.

In response to the second fatality, BMO Capital downgraded Sarepta's stock. The company is now exploring potential changes to the immunosuppressive regimen used alongside the treatment. Additionally, Sarepta has suspended shipments of Elevidys for non-ambulatory patients and paused the ENVISION study, which is evaluating the therapy in older ambulatory and non-ambulatory individuals with Duchenne.

Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. The company is working to convene an independent group of experts to consider an enhanced immunosuppression regimen for Elevidys. This panel will evaluate data and assess the proposed regimen, which includes sirolimus and is supported by preclinical data. Sarepta will share the panel's recommendations with the FDA, and implementation of any new regimen will be subject to FDA guidance and allowance.