Roche announced a new global Phase 3 trial for Elevidys (delandistrogene moxeparvovec), seeking a path toward European approval after a negative European Medicines Agency review. The study is designed to generate additional placebo-controlled evidence on motor outcomes over 72 weeks in early ambulatory Duchenne patients. Roche said it will enroll roughly 100 patients and use a “time to rise” measure as the primary endpoint, reflecting the regulator’s earlier reservations about linking the therapy’s biological effect to clinically meaningful functional benefit. The move follows complications and restrictions in the US market. The planned trial signals how gene-therapy approval strategies are shifting toward more targeted endpoints and additional evidence packages when regulators question clinical translation.