Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001
05/24/23 8:00 AM EDT
New regulatory action date is
- 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 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.
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.
Sarepta will remain in a quiet period for the duration of the BLA review.
About SRP-9001 (delandistrogene moxeparvovec)
SRP-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
Sarepta is on an urgent mission: To engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.
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This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, business plans, priorities, research and development programs; a potential accelerated approval for SRP-9001 for 4-5 year olds; the potential for an expansion of the SRP-9001 label upon completion and FDA review of EMBARK; the potentially transformative benefits of SRP-9001; and plans and milestones, including top-line results for EMBARK in the fourth quarter of 2023 and the anticipated regulatory action date of
Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: the FDA may not approve the BLA for SRP-9001 by
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