Sarepta Therapeutics shares added almost 25% on Monday morning after the US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee voted eight to six in favor of the accelerated approval of the company’s therapeutic SRP-9001 for Duchenne muscular dystrophy (DMD).
The company said the vote, while not binding, would be considered by the FDA when considering the potential accelerated approval of SRP-9001.
SRP-9001 (delandistrogene moxeparvovec) is a gene transfer therapy designed to address the underlying cause of DMD through the targeted production of functional components of dystrophin in muscle tissue, according to Sarepta.
The Biologics License Application (BLA) for SRP-9001 is currently under priority review by the FDA with a regulatory action date of May 29, 2023.
“Today’s advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies,” said Sarepta CEO Doug Ingram in a statement on May 12.
“With the May 29 action date our top priority, we will work collaboratively with the FDA to complete the review of our BLA for SRP 9001.
“We extend our sincere appreciation to the families, clinicians, FDA presenters and committee members who participated in today’s panel and to all those who provided input and comments both in the written record and in the open public hearing.”
Sarepta shares surged 24.9% to US$150.07 in New York on Monday morning.
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