European Regulators Reject Duchenne Muscular Dystrophy Treatment Elevidys

European drug regulators have recommended rejecting Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), in a setback for its developers, Roche and Sarepta Therapeutics. The European Medicines Agency (EMA) found that Elevidys did not significantly improve movement in children aged 3 to 7 who can still walk, despite producing a shortened version of the muscle-protecting protein dystrophin. The study also failed to show benefits in a subgroup of patients who were expected to respond better to treatment. While the EMA did not comment on safety, Elevidys has been linked to rare but serious liver complications, including deaths in older patients. Roche plans to work with regulators to explore alternative approval paths, citing unmet treatment needs for DMD. However, experts believe overturning the decision will be difficult. This rejection adds to recent challenges for Sarepta, which has already faced U.S. distribution halts due to safety concerns and potential financial struggles. Elevidys was controversially approved in the U.S. in 2023, but its future remains uncertain. Without European approval, Sarepta loses potential revenue and faces growing financial pressure.

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