Sarepta Gets FDA Priority Review for SRP-9001 in Duchenne Muscular Dystrophy
November 28 2022 - 8:01AM
Dow Jones News
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 colin.kellaher@wsj.com
(END) Dow Jones Newswires
November 28, 2022 07:46 ET (12:46 GMT)
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