The first DMD gene therapy SRP-9001 was delayed by the FDA for 3 week
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The first DMD gene therapy SRP-9001 was delayed by the FDA for 3 weeks, and it is only used for patients aged 4-5.
The first gene therapy for Duchenne muscular dystrophy (DMD), Sarepta’s SRP-9001 (delandistrogene moxeparvovec), in hopes of securing a landmark decision by the U.S. Food and Drug Administration (FDA) by May 29, will have to wait another About three weeks later.
On May 24, Sarepta issued an announcement stating that after discussions with the FDA, the bureau stated that the FDA needs moderate additional time to complete the review, including final label negotiations and post-marketing commitment discussions, and is expected to review the biological status of SRP-9001.
The product license application (BLA) will be completed by June 22, 2023 .
And, after completing the review, the FDA is working towards accelerated approval of SRP-9001, though initially only for DMD patients aged 4-5 .
EMBARK is a global randomized, double-blind, placebo-controlled Phase 3 trial of SRP-9001, the proposed confirmatory study.
The FDA has informed Sarepta that, in addition to confirming the initial BLA approval results, the agency intends to conduct a non-age-restricted label extension for SRP-9001 based on a review of the EMBARK data if the trial meets its goals.
EMBARK is fully enrolled and topline results are expected in the fourth quarter of 2023.
Previous news:
FDA Questions Efficacy and Safety of DMD Gene Therapy SRP-9001
FDA Advisory Committee Meeting on the first DMD gene therapy SRP-9001 is set for May 12th
The first DMD gene therapy SRP-9001 may cost 4 million US dollars
DMD gene therapy phase 2 clinical trials are not effective
(source:internet, reference only)
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