Today, Sarepta Therapeutics shared an update through a community letter regarding its research and clinical development programs, including an important decision about the Phase 3 ENVISION clinical trial evaluating ELEVIDYS in non-ambulatory and late-ambulatory individuals living with Duchenne.

The letter also highlighted new ELEVIDYS findings presented at recent scientific meetings, including the 2026 World Muscle Society (WMS) Congress, and reiterated that the FDA’s target decision date for Sarepta’s applications seeking traditional approval of AMONDYS 45 and VYONDYS 53 is expected at the end of February 2027.

ENVISION Update

ENVISION was designed to evaluate ELEVIDYS in non-ambulatory and late-ambulatory individuals with Duchenne, enrolling 90 participants across 14 countries (64 non-ambulatory and 26 late-ambulatory), and has been on global hold for over one year. After considering the length of the hold and additional safety information needed to evaluate treatment in non-ambulatory individuals, Sarepta determined that restarting the study would require significant changes to its original design and is ultimately not feasible. The company will instead close enrollment and analyze the blinded data collected in Part 1, with the goal of sharing findings with the community in a more timely manner.

The decision to close ENVISION is disappointing news for the individuals and families who have been following and participating in the trial. We recognize the hope, time, and commitment that families have invested in this research, and understand that this update may bring difficult questions and uncertainty. 

According to Sarepta, clinical trial sites are working directly with participants and families to discuss individual next steps and transition plans. If you or a family member is participating in ENVISION, please contact your study investigator or clinical trial team with questions about your participation and follow-up. Individuals and families may also contact advocacy@sarepta.com.

ENDEAVOR Cohort 8

Sarepta emphasized that its commitment to exploring a potential treatment path for non-ambulatory individuals remains steadfast. The company continues to enroll non-ambulatory participants in ENDEAVOR Cohort 8, which is evaluating an enhanced safety approach using oral sirolimus alongside corticosteroids. Findings will help inform safety considerations and next steps, with Sarepta planning to engage with the FDA in Q1 2027 to discuss the data and a potential path forward for non-ambulatory individuals in the U.S.

Our commitment is to provide clear, accurate, and trusted information to help make sense of what these updates mean for our community, what we know, what questions remain, and what may come next. PPMD will continue to follow developments from Sarepta, including findings from ENDEAVOR Cohort 8 and upcoming discussions with the FDA. As always, we will share meaningful updates with the Duchenne community as additional information becomes available.

Read Sarepta’s community letter here.