Roche announced a new global Phase 3 trial for Elevidys on April 16, 2026, following the European Medicines Agency's (EMA) rejection last year due to insufficient evidence of benefit in ambulatory DMD patients.123
The trial will enroll about 100 early ambulatory boys, comparing Elevidys to placebo over 72 weeks, with primary endpoint of change in 'time to rise from floor'.126
EMA's negative opinion was based on the EMBARK study failing its primary endpoint, despite some secondary improvements and longer-term data.14
Elevidys is approved in the US and several countries for ambulatory DMD boys; Roche holds rights outside the US.123
Roche aims for this trial to support resubmission for EU approval and reimbursement elsewhere, citing confidence from long-term data in over 1,000 patients.12
Sources:
1. https://www.packgene.com/frontier/041626-roche/
2. https://www.biopharmadive.com/news/roche-elevidys-europe-trial-duchenne-gene-therapy-sarepta/817674/
3. https://www.statnews.com/2026/04/16/roche-elevidys-pivotal-trial-europe-sarepta/
4. https://globalgenes.org/raredaily/chmp-rejects-sareptas-elevidys/