Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) plunged 13% on Friday after a major regulatory blow in Europe. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on Elevidys, the company’s gene therapy designed to treat Duchenne muscular dystrophy.
The CHMP declined to support approval of Elevidys for ambulatory children aged 3 to 7 years, the age group targeted in the application. The decision further complicates Sarepta’s efforts to bring the therapy to global markets. Outside of the U.S., Elevidys is marketed by Roche Holding AG (SIX:ROG), which had been seeking authorization for use in Europe to treat the rare and progressive neuromuscular condition.
This latest setback follows a recent move by the U.S. Food and Drug Administration, which requested a pause in Elevidys’ use in the American market. Earlier this week, Sarepta confirmed it had temporarily halted U.S. shipments in response.
Roche has also suspended distribution of the treatment in markets that rely on FDA decisions, significantly narrowing Elevidys’ potential commercial footprint. Roche had taken the lead on commercialization outside the U.S. under a partnership agreement with Sarepta.
Duchenne muscular dystrophy is a rare, fatal genetic disorder that primarily affects boys, leading to muscle wasting, severe mobility impairment, and shortened life expectancy.
The regulatory setbacks in both the U.S. and EU now pose serious hurdles to Sarepta’s rollout strategy for Elevidys, which had been viewed as a potential breakthrough for patients facing a disease with few effective treatment options.
Sarepta Therapeutics stock price
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