Press release
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001
New regulatory action date is June 22, 2023 CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"\nNew regulatory action date is June 22, 2023\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today 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.\n\n\n\nFollowing 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.\n\n\n\nEMBARK, the global, randomized, double-blind, placebo-controlled Phase 3 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.\n\n\n\nThe 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.\n\n\n\nSarepta will remain in a quiet period for the duration of the BLA review.\n\n\nAbout SRP-9001 (delandistrogene moxeparvovec)\nSRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy designed to address the underlying cause of Duchenne through the targeted production of functional components of dystrophin in muscle tissue. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to combine Roche’s global reach, commercial presence and regulatory expertise with Sarepta’s gene therapy candidate for Duchenne to accelerate access to SRP-9001 for patients outside the United States.\n\n\nAbout Sarepta Therapeutics\nSarepta is on an urgent mission: To engineer precision genetic medicine for rare diseases ...