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April 16, 2026 — Roche said it will initiate a global, placebo-controlled Phase III study of Elevidys, the gene therapy for Duchenne muscular dystrophy (DMD), after feedback from the European Medicines Agency.
The trial will enrol about 100 early ambulatory boys with confirmed DMD mutations and compare Elevidys to placebo over more than 72 weeks, with the primary endpoint the change in “time to rise” from the floor.
Roche, which holds rights to Elevidys outside the United States, said the study is intended to generate the additional data needed for a European re-submission.
Elevidys, developed by U.S. firm Sarepta Therapeutics and approved in nine countries, faced a negative EMA opinion last year after questions about clinical benefit and two patient deaths that prompted a temporary U.S. shipment pause and FDA restrictions.
Roche cited long-term data and experience treating over 1,000 ambulatory boys.
The move follows sales declines for Elevidys — roughly $900 million net product revenue in 2025 — and market setbacks that have reduced its earlier multi-billion-dollar forecasts.








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