Sarepta Gets FDA Priority Review for SRP-9001 in Duchenne Muscular Dystrophy

By Colin Kellaher

Sarepta Therapeutics Inc. on Monday said the U.S. Food and Drug Administration granted priority review to its application seeking accelerated approval of SRP-9001 for the treatment of ambulant individuals with Duchenne muscular dystrophy.

The Cambridge, Mass., genetic-medicine company said SRP-9001, if approved, would be the first gene therapy for Duchenne, a degenerative and invariably fatal disease that robs children of muscle and function.

The FDA grants priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period. Sarepta said the agency has set a target action date of May 29, 2023, for the application.

Sarepta is developing SRP-9001 with Swiss pharmaceutical company Roche Holding AG under a 2019 partnership that included an upfront payment of more than $1 billion.

Duchenne is characterized by a mutation in the dystrophin gene that results in the lack of dystrophin, which acts as a shock absorber for muscle at the membrane. SRP-9001 is a one-time treatment designed to treat the underlying cause of Duchenne by delivering a functional shortened dystrophin to muscle.

Shares of Sarepta, which closed Friday at $114.57, rose 5.4% to $120.74 in premarket trading.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

November 28, 2022 07:46 ET (12:46 GMT)

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