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Sarepta announces FDA grants priority review for SRP-9001 BLA
The Fly

Sarepta announces FDA grants priority review for SRP-9001 BLA

Sarepta Therapeutics announced that the U.S. Food and Drug Administration has accepted the Company’s Biologics License Application seeking accelerated approval of SRP-900 for the treatment of ambulant individuals with Duchenne muscular dystrophy. SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche. 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 designed to treat the proximate cause of Duchenne by delivering to muscle a gene that codes for a shortened, functional form of dystrophin. In addition to a wealth of pre-clinical evidence, the BLA for SRP-9001 included efficacy and safety data from Study SRP-9001-103, as well as from Studies SRP-9001-101 and SRP-9001-102, and an integrated analysis across these three clinical studies comparing functional results to a propensity-score-weighted external control. In clinical results from more than 80 treated patients, SRP-9001 has demonstrated positive results at multiple time points, including one-, two- and up to four-years after treatment, in addition to demonstrating a consistent safety profile. In addition to Studies SRP-9001-101, SRP-9001-102 and SRP-9001-103, SRP-9001 is also being studied in EMBARK, a global, randomized, double-blind, placebo-controlled clinical trial of SRP-9001 which has recruited 125 participants with Duchenne between the ages of 4 to 7. EMBARK is fully enrolled with results expected by the end of 2023. Sarepta has proposed EMBARK as the post-marketing confirmatory trial for SRP-9001.

Published first on TheFly

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