Sarepta Therapeutics Inc. surged the most since late 2019 after US regulatory advisers recommended approval for the company’s gene therapy for a severe inherited muscular disease.
(Bloomberg) — Sarepta Therapeutics Inc. surged the most since late 2019 after US regulatory advisers recommended approval for the company’s gene therapy for a severe inherited muscular disease.
The advisers voted 8-6 Friday evening in favor of clearing Sarepta’s genetic treatment for Duchenne muscular dystrophy, a condition that mainly affects boys, killing most by their early 20s. The Food and Drug Administration isn’t required to follow the recommendations of its advisers, although it often does. Sarepta shares rose as much as 26% at the market open in New York their biggest intraday gain since December 2019.
Following the vote, the full FDA is highly likely to approve the therapy by a May 29 deadline, given the positive outcome and the relatively benign safety profile for the drug, Cowen analyst Ritu Baral wrote in a note to clients.
Expert panelists were split, with two patient and consumer representatives tipping the vote in favor of Sarepta’s therapy. The treatment has shown in studies that it raises levels of a protein believed to indicate patient benefit, but those same tests haven’t shown actual improvements in health compared to placebo. Nonetheless, the recommendation is a huge win for Sarepta, likely paving the way for the company’s fourth accelerated approval for a Duchenne drug and the first for a gene therapy for the disease.
“The vote was ultimately positive as panelists believed risks were manageable and patients should be allowed to have access to drug,” Citigroup Inc. analysts led by Neena Bitritto-Garg wrote in a note to clients. They have a buy rating on the shares.
(Updates with analyst comment in third paragraph)
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