Sarepta Therapeutics has decided not to resume enrollment in ENVISION, the Phase 3 confirmatory trial of its Duchenne muscular dystrophy gene therapy Elevidys in non ambulatory and late ambulatory patients. The company said the study has been on global hold for more than a year, and that after considering the length of the hold and the additional safety information needed in non ambulatory patients, restarting would require significant changes to the original design and is ultimately not feasible.
ENVISION enrolled 90 participants across 14 countries, 64 of them non ambulatory and 26 late ambulatory. Sarepta will close enrollment and analyse the blinded data collected in Part 1 of the study, with the goal of sharing findings with the community sooner than a restarted trial would allow. The decision was confirmed on the company’s third quarter earnings call on November 6.
The trial’s history is the therapy’s safety history. Elevidys shipments to non ambulatory patients were suspended after fatal cases of acute liver failure, and in November 2025 the FDA removed the non ambulatory indication from the label and added a boxed warning for acute liver failure, while ambulatory patients retained traditional approval. ENVISION was the confirmatory trial underpinning the accelerated approval for the non ambulatory population, so its closure removes the study that was meant to verify clinical benefit in that group.
Sarepta’s remaining route back runs through ENDEAVOR Cohort 8, which continues to enroll non ambulatory participants and evaluates an enhanced safety approach using oral sirolimus alongside corticosteroids, aimed at mitigating the liver injury risk. The company plans to engage with the FDA in the first quarter of 2027 to discuss the data and a potential path forward for non ambulatory patients in the United States. Separately, the company noted that the FDA target decision date for its applications seeking traditional approval of Amondys 45 and Vyondys 53 is expected at the end of February 2027.