Sarepta is preparing a second Phase 3 registration-enabling attempt for Elevidys after EMA denied marketing authorization, pushing the gene-therapy timeline back for Duchenne muscular dystrophy access in Europe. The European regulator cited that the initial evidence set did not meet efficacy and safety requirements, including a lack of significant movement benefit at 12 months under conditional approval. US FDA approval remains in place, but the reassessment reflects how regional evidence thresholds can differ. Reporting also notes that Roche, the licensing partner, will oversee the new Phase III trial setup to address EMA’s concerns using additional placebo-controlled efficacy and safety data over 72 weeks.