FDA changes course on Sarepta AdComm meeting

March 17, 2023

In a surprising turn of events, Sarepta Therapeutics announced this week that the FDA will be holding an AdComm meeting after all for its Duchenne muscular dystrophy (DMD) gene therapy candidate.

The agency initially accepted SRP-9001’s BLA in November of last year and gave it a regulatory action date of May 29, 2023. According to Sarepta’s recent statement, the FDA’s decision to hold an AdComm meeting is “a change from the communicated position during the midcycle meeting,” but something the biotech has been preparing for since the submission of the BLA. 

Since Sarepta’s gene therapy has one of the first BLAs founded on surrogate endpoints, the meeting will likely discuss the totality of evidence that helps support the premise that SRP 9001 dystrophin is reasonably likely to predict clinical benefit. 

SRP-9001 is being developed in partnership with Roche. Back in Dec. 2019, Roche handed over $1.15 billion to obtain the rights to the drug outside the U.S. Recently, Sarepta signed a commercial supply agreement with CDMO giant Catalent to manufacture the drug.