Roche announced it will initiate another global Phase 3 trial for Duchenne muscular dystrophy gene therapy Elevidys, aiming to generate data that could support an updated European resubmission. The move follows a negative European Medicines Agency assessment in 2025, where regulators questioned whether testing showed meaningful long-term motor benefits and whether Elevidys’ biological effect could be linked to clinical outcomes. Roche said the new study will enroll about 100 early ambulatory boys and compare Elevidys with placebo over 72 weeks, using “time to rise” as a key endpoint tied to disease progression. This is Roche’s second attempt to turn the Elevidys evidence package into a regulatory path in Europe, after prior setbacks that also included restrictions in the U.S. and controversy surrounding safety events.