SAREPTA THERAPEUTICS, INC. By Colin Kellaher
Sarepta Therapeutics Inc. on Friday said it plans to file for U.S. Food and Drug Administration accelerated approval of its SRP-9001 gene therapy to treat ambulant individuals with Duchenne muscular dystrophy.
The Cambridge Mass., genetic-medicine company said it expects an FDA review to begin this year and run through the first half of 2023.
Duchenne muscular dystrophy is a rare, fatal neuromuscular genetic disease that occurs in roughly one in every 3,500 to 5,000 males.
The FDA in 2020 granted fast-track designation, which aims to facilitate the development and expedite the review of treatments for serious or potentially life-threatening illnesses with high unmet medical needs, to SRP-9001, which has also received the agency's rare-pediatric-disease and orphan-drug designations.
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.
Trading in shares of Sarepta, which closed Thursday at $85.93, was halted premarket on Friday.
Write to Colin Kellaher at colin.kellaher@wsj.com
(END) Dow Jones Newswires
07-29-22 0734ET
