Sarepta moved to restart its path in Europe for Duchenne muscular dystrophy gene therapy Elevidys after the EMA declined marketing authorization following inadequate efficacy evidence. The company will launch an additional Phase III trial designed to address the regulator’s concerns, while Roche—Sarepta’s partner—will oversee the registration-enabling study. The new trial is structured around placebo-controlled efficacy and safety data over 72 weeks, with primary results expected after enrollment and study execution. The move follows prior issues for Elevidys in both regulators and patients, including reported acute liver failure deaths tied to dosing in 2025 and earlier FDA-related shipment pauses.