Sarepta Therapeutics (SRPT) announced AdCom to be held for SRP-9001
Sarepta Therapeutics, Inc. (SRPT) announced that at its late cycle meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA), the U.S. Food and Drug Administration’s Office of Therapeutics (OTP) has determined that an advisory committee meeting will be held for SRP-9001 in advance of the May 29, 2023 regulatory action date. SRP-9001 is Sarepta’s investigational gene therapy for the treatment of Duchenne muscular dystrophy.
“FDA’s decision to hold a public advisory committee meeting on the SRP 9001 BLA is a change from the communicated position at the midcycle meeting. FDA leadership has noted publicly that FDA is interested in exploring the use of surrogate endpoints, biomarkers, and innovative approaches like accelerated approval to advance cell and gene therapies, particularly for rare, life-ending degenerative diseases. It is our understanding that as one of the first gene therapy BLAs founded on a surrogate endpoint, the advisory committee will primarily relate to the totality of evidence supporting the conclusion that the SRP 9001 dystrophin is reasonably likely to predict clinical benefit, the standard for accelerated approval,” said Doug Ingram, president and chief executive officer, Sarepta. “While we are disappointed that we must communicate a change in decision after our prior statement on the topic, we are not disappointed with the decision to hold an advisory committee. We had been preparing for an advisory committee meeting from the filing of the BLA in the fall of 2022. We will be well prepared, and look forward to presenting the wealth of evidence supporting the transformative potential of SRP-9001. We would like to thank CBER for moving expeditiously to schedule the advisory committee in advance of our May 29, 2023, regulatory action date, once the change in decision was made.”
Conference call details
At 4:30 p.m. ET on March 16, 2023, Sarepta will host a conference call and webcast to discuss this update.
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