FDA accepts filing for SRP 9001 and gives it priority review for Duchenne muscular dystrophy
Sarepta Therapeutics announced that the FDA has accepted the Company's Biologics License Application (BLA) seeking accelerated approval of SRP 9001 (delandistrogene moxeparvovec) 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.
The BLA for SRP 9001 included efficacy and safety data from Study SRP-9001-103 (also known as ENDEAVOR), 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 (EC). 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.
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