Sarepta announced it is launching an additional Phase 3 trial for Elevidys (delandistrogene moxeparvovec) to address European regulator concerns after the EMA denied marketing authorization last year. Roche, which licensed the therapy, will oversee the new registration-enabling study, designed to generate additional placebo-controlled efficacy and safety data over 72 weeks. The EMA’s 2025 decision cited insufficient evidence in the initial conditional authorization evidence package, including no significant effect on movement abilities at 12 months in the EMBARK program. Sarepta and Roche aim to generate the data they believe meet EMA regulatory requirements for approval in Europe. The company also referenced prior safety scrutiny in the program. Following two patient deaths attributed to acute liver failure in non-ambulatory recipients, the FDA requested a voluntary halt to shipments, and dosing was suspended for non-ambulatory patients in clinical and commercial settings. For the Duchenne gene therapy market, the move keeps Elevidys in play in Europe but highlights the fragility of regulatory pathways when benefit trajectories depend on timepoints, endpoint selection, and placebo-controlled designs.