Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure

CureDuchenne is sad to share that another person has passed away due to acute liver failure after treatment with Elevidys, Sarepta’s micro-dystrophin gene therapy.  Our hearts go out to the family and friends of this individual at this difficult time.  

While regulators and experts are reviewing the data and looking at possible safety enhancements including additional immunosuppression, Sarepta will be pausing Elevidys treatments for non-ambulatory individuals.  Sarepta is also pausing the ENVISION Study in older ambulatory and non-ambulatory individuals.  While ENVISION is complete in the U.S., it was still screening and dosing at ex-US sites. 

Sarepta disclosed the first death, which was a 16-year-old boy, earlier this year. 

Press release HERE

Letter to the Community: