Alarm over Elevidys patient death

19 March 2025

Genetic medicine firm Sarepta Therapeutics (Nasdaq: SRPT) announced the tragic death of a young man with Duchenne muscular dystrophy (DMD) following treatment with the company’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl).

The patient suffered acute liver failure, a known possible side effect of Elevidys and other AAV-mediated gene therapies that is highlighted in the prescribing information.

In a statement, Sarepta said that, although it is not a new safety signal and the benefit-risk of Elevidys remains positive, a case 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.

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