Sarepta Therapeutics, Inc.
NEWS
The FDA’s CBER approved nine novel therapies in the first half of 2023, with several more high-profile decisions on deck for the second half of the year.
Shares of Sarepta dropped 11% a day after securing accelerated approval for the first gene therapy to treat Duchenne muscular dystrophy over concerns about the potential for label expansion.
The FDA has four target action dates this week, including ones for Argenx’s subcutaneous efgartigimod in generalized myasthenia gravis and Sarepta’s gene therapy for Duchenne muscular dystrophy.
The extensive discussion between the FDA’s advisory committee, company representatives and other interested parties could serve as a cautionary tale to developers of future gene therapies.
The FDA set a new action date of June 22 for Sarepta’s gene therapy for the neuromuscular disease, approximately three weeks after the original date of May 29.
The FDA’s agenda includes an advisory committee meeting for Sarepta’s delandistrogene moxeparvovec, which, if approved, would become the first gene therapy for Duchenne muscular dystrophy.
Peter Marks described the steps the agency is taking to advance the development of gene therapies for rare disorders. This could spell good news in the near future for Sarepta Therapeutics.
The FDA will hold an advisory committee meeting for Sarepta’s investigational gene therapy for Duchenne muscular dystrophy ahead of its May 29, 2023 action date.
The FDA has accepted Sarepta’s BLA for the accelerated approval of SRP-9001, an investigational gene therapy for DMD. Roche is responsible for commercialization outside the U.S.
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