FDA to hold AdCom on Sarepta's SRP-9001 after all

US precision genetic medicine for rare diseases developer Sarepta Therapeutics (Nasdaq: SRPT) yesterday saw its shares fall 20% in after-hours trading, as it revealed that, at its late cycle meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA), the US Food and Drug Administration’s Office of Therapeutics (OTP) has determined that an advisory committee meeting will be held for the drug candidate in advance of the May 29, 2023 regulatory action date.

This is a change of heart at the US regulator, as earlier this month Sarepta said that the FDA would not be requiring an advisory committee meeting prior to the PDUFA date.

SRP-9001 is Sarepta’s investigational gene therapy for the treatment of Duchenne muscular dystrophy. The BLA was accepted for priority review by the FDA in November last year. It would be Sarepta’s fourth DMD drug to reach market based on early or biomarker data.