Sarepta to seek early FDA approval for gene therapy to treat Duchenne muscular dystrophy

Sarepta Therapeutics said Friday that it intends seek regulators’ approval for its gene therapy to treat Duchenne muscular dystrophy earlier than expected — a risky move but one that could help the company reach the market much faster.

In early Friday trading, Sarepta shares rose 12% to $96.50.

Sarepta is asking the Food and Drug Administration to approve the one-time treatment called SRP-9001 based on its ability to produce large increases in a crucial muscle protein typically missing in children born with Duchenne. Without dystrophin, muscles break down over time, which is why Duchenne patients gradually lose their ability to walk and breathe. The disease is fatal, often by the time boys are in their late teens or early 20s.

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