Sarepta Therapeutics announces update on FDA regulatory review of SRP 9001
Sarepta Therapeutics, Inc. provided the following update on the Biologics License Application (BLA) for SRP 9001 (delandistrogene moxeparvovec), which is currently under review for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the DMD gene
Following discussions with FDA, the Agency has indicated that, subject to the completion of the BLA review, it is working toward potentially granting an accelerated approval for SRP 9001, initially for use in Duchenne patients ages 4-5 years old. EMBARK, the global, randomized, double-blind, placebo-controlled Phase III trial of SRP 9001, is the proposed confirmatory study.
The Agency has informed Sarepta that, in addition to confirming the results of the initial BLA approval, if the trial meets its objectives the Agency intends to entertain a non-age-restricted expansion of the SRP 9001 label based upon the review of the EMBARK data. EMBARK is fully enrolled, with top-line results expected in the fourth quarter of 2023.
The FDA has also informed Sarepta that it requires modest additional time to complete the review, including final label negotiations and postmarketing commitment discussions, and that it anticipates that the review will be complete by June 22, 2023.
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