Sarepta Therapeutics, Inc.
NEWS
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.
The World Muscle Society 2022 Congress is ongoing in Halifax, Canada, with numerous companies presenting cutting-edge research in neuromuscular diseases. Here’s a look.
The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
Sarepta Therapeutics and Alnylam Pharmaceuticals have announced the pricing of $1 billion and $900 million in convertible senior notes respectively.
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