Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval

Two Sarepta Therapeutics drugs for treating different genetic subsets of patients with Duchenne muscular dystrophy failed the confirmatory study required of their accelerated FDA approvals. Sarepta executives say there’s still a path for traditional FDA approval, but it might rely on regulatory flexibility that the agency has shown toward the company and other developers of therapies for rare diseases.

Sarepta’s Vyondys 53, approved in 2019 , and Amondys 45, approved in 2021 , both received their speedy regulatory nods based on clinical trial results showing treatment led to higher levels of dystrophin, a key muscle protein that Duchenne patients lack. But these antisense oligonucleotide drugs were still required to undergo testing in a longer and larger confirmatory study designed