Sarepta to continue shipping gene therapy despite patient deaths

Sarepta Therapeutics said it will continue shipping its gene therapy Elevidys for Duchenne muscular dystrophy patients despite an FDA request to pause distribution following multiple patient deaths. 

The FDA’s request July 18 came after Sarepta confirmed the death of a third patient from acute liver failure tied to its gene therapy programs. 

The drugmaker said the death involving a 51-year-old patient occurred in a separate early stage trial for a different gene therapy, not Elevidys, according to a July 18 news release from the company. However, two deaths earlier in 2025 were linked directly to Elevidys, which is the only FDA-approved gene therapy for Duchenne. 

Citing its own analysis of safety data, Sarepta said there were no new or changed safety signals in the ambulant patient population and that it will continue to ship the medication to those patients.