As Sarepta loses platform designation, pathway’s future is uncertain

The first platform technology designation issued by FDA has been revoked, validating earlier concerns about its issuance and raising questions about whether a suitable use case for designation exists at this time. 

One of the latest shoes to drop in the saga of Sarepta Therapeutics Inc. (NASDAQ:SRPT) is FDA’s revocation of the platform technology designation granted for the company’s AAVrh74 vector to treat limb girdle muscular dystrophy (LGMD). Announced Friday, alongside clinical holds on all of Sarepta’s LGMD gene therapy trials, the decision follows the death of a third trial patient treated with the vector from acute liver failure...