Biologics

Sarepta BLA Needs Panel Review: FDA

FDA has told Sarepta Therapeutics that it has decided to hold an advisory committee meeting to review its gene therapy BLA for SRP-9001 (delandistrogene moxeparvovec), indicated for treating Duchenne muscular dystrophy. Previously when the submission was accepted for review, the agency said that an advisory committee meeting was not necessary. The committee meeting is expected to be held before the 5/29 user fee review action target date.

“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,” the company explains. “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.”

SRP-9001 is an investigational gene therapy being developed in partnership with Roche. It is designed to deliver therapy to muscle tissue for the targeted production of functional components of dystrophin, according to the company.

DMD is a rare, fatal neuromuscular genetic disease that occurs in about one in every 3,500-5,000 newborn males worldwide, Sarepta says. It is caused by a change or mutation in the gene that encodes instructions for dystrophin. Those with the disease eventually face increasing difficulty in breathing due to respiratory muscle dysfunction, and cardiac dysfunction can lead to heart failure, it says.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome