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Investing.com -- Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock declined 13% Friday after European regulators recommended against approving Elevidys, the company’s gene therapy for Duchenne muscular dystrophy.
The European Medicines Agency’s drug advisory committee (CHMP) rejected Elevidys for children aged 3 to 7 years who are able to walk, the agency announced Friday. The therapy, which is marketed outside the U.S. by Roche Holding AG (SIX:ROG), was seeking approval for treating the rare genetic disease that causes progressive muscle weakness.
This setback follows closely on the heels of the U.S. Food and Drug Administration’s recent request for a pause on Elevidys use in the United States. Sarepta had already announced a temporary halt on U.S. shipments earlier this week.
Roche has also paused shipments in jurisdictions that reference FDA approvals, further limiting the therapy’s market reach. The Swiss pharmaceutical giant had been responsible for commercializing the treatment in markets outside the U.S. after partnering with Sarepta.
Duchenne muscular dystrophy is a rare genetic disorder that primarily affects boys and leads to progressive muscle deterioration. The condition is ultimately fatal, with patients typically experiencing serious mobility limitations and shortened lifespans.
The dual regulatory setbacks in both the U.S. and European markets represent significant challenges for Sarepta’s commercialization strategy for Elevidys, which had been positioned as a breakthrough treatment option for the debilitating condition.