FDA AdComm backs Sarepta Duchenne gene therapy

May 15, 2023

Last Friday, in a narrow 8-6 vote, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted to back the approval of Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy.

Duchenne is caused by a mutation in the Dystrophin gene, so SRP-9001 is designed to deliver the microdystrophin-encoding gene directly to the muscle tissue for the targeted production of the microdystrophin protein. During Friday’s hearing, committee members expressed apprehension regarding the treatment’s usage of engineered viruses for delivery, sharing concern over alternative treatments for patients if Sarepta's therapy were to be deemed ineffective in the future.

Documents released ahead of the meeting stated that the FDA “notes that the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with DMD,” adding that the agency had “safety concerns related to the possibility of administering an ineffective gene therapy."

The agency first accepted Sarepta’s BLA back in November, granting it Priority Review and assigning a PDUFA date of May 29, 2023. Initially, no ADComm meeting was planned, but back in March, Sarepta announced that the FDA would in fact be holding an AdComm meeting after all.

SRP-9001 — a driving force behind Roches $1.5 billon acquisition of Sarepta in 2019 — is slated to be manufactured by Catalent should it win approval.