Sarepta Therapeutics Shares Safety Update on ELEVIDYS

CAMBRIDGE, Mass., March 18, 2025 -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following safety update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.

We are profoundly saddened to share that a young man with Duchenne muscular dystrophy has passed away following treatment with ELEVIDYS, having suffered acute liver failure. Acute liver injury is a known possible side effect of ELEVIDYS and other AAV-mediated gene therapies and is highlighted in the prescribing information. Although it is not a new safety signal and the benefit-risk of ELEVIDYS remains positive, acute liver failure (ALF) leading to death represents a severity of acute liver injury not previously reported for ELEVIDYS, which to date has been used to treat more than 800 patients in clinical trials or as a prescribed therapy.

In addition, testing revealed this patient had a recent cytomegalovirus (CMV) infection which was identified by the treating physician as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis