Roche and Sarepta Update on ELEVIDYS Study 

Roche and Sarepta Update on ELEVIDYS Study 

This week Roche and Sarepta Therapeutics have shared updates on the ongoing development programme for ELEVIDYS (delandistrogene moxeparvovec). 

The companies have decided to close recruitment to the global Phase 3 ENVISION study and proceed with analysis of the blinded data already collection from 90 participants across 14 countries. The study has been on a global clinical hold since 2025 following two cases of acute liver failure in non-ambulatory participants 

The decision follows challenges in restarting the trials after its prolonged pause. Potential changes to the treatment approach could make the original study results more difficult to interpret. Researchers continue to evaluate approaches that may help reduce risk in non-ambulatory individuals, including the use of immunosuppressive agenda in the ongoing ENDEAVOR study.  

Participants enrolled in ENVISION are being contacted by their study teams regarding next steps. Individuals who receive ELEVIDYS may be invited to enter the long-term follow-up study, EXPEDITION, while the possibility of cross opportunity for participants who receive placebo remains under evaluation and would require supportive data and regulatory approval.  

Roche has also confirmed that planning continues for a new global Phase 3 study in ambulatory boys, with further information expected once recruitment is ready to open. In addition, Sarepta Therapeutics shared new data from its ELEVIDYS and exon-skipping programmes and reiterated its commitment to exploring a potential development pathway for non-ambulatory individuals living with Duchenne.  

You can read Sarepta’s community letter here:

and Roche’s community letter here. 

If you have any questions or concerns about your treatment, please speak with your specialist Duchenne care team. If you would like a wider conversation following this news, please give us a call on 07535 498 506 or email us at support@actionduchenne.org. If you have any questions about the science, research, or the information shared in this update, please feel free to contact our Science Team at science@actionduchenne.org, who will be happy to support you. 

We recognise that treatment updates can sometimes feel worrying. If you have concerns, your specialist clinical team remains the best source of advice for your individual circumstances.