Roche Restarts Phase III Trial to Pursue EMA Approval for Elevidys

Roche is initiating a new Phase III trial for Elevidys, its gene therapy for Duchenne muscular dystrophy, after the European Medicines Agency declined to approve the treatment last year. The EMA rejection followed scrutiny tied to two patient deaths connected to the therapy, and the new study is designed specifically to generate the placebo-controlled data required for a regulatory re-submission in Europe. Elevidys, developed in partnership with Sarepta Therapeutics, is currently approved in nine countries for ambulatory boys with a confirmed DMD mutation. In the United States, shipments were temporarily paused last July before resuming. The therapy has now been administered to more than a thousand ambulatory boys worldwide, according to Roche Chief Medical Officer Levi Garraway. The new trial will enroll approximately 100 early ambulatory boys with DMD and run for more than 72 weeks. The primary endpoint is change in time-to-rise from the floor velocity, a functional measure relevant to disease progression. Roche is betting that placebo-controlled efficacy data will satisfy the EMA's outstanding evidentiary requirements. A successful re-submission would open the substantial European rare-disease market to Elevidys and reduce Roche's geographic revenue concentration. The key watch point is whether the trial's safety profile, under renewed scrutiny after the prior deaths, holds across the expanded cohort.