Sarepta Therapeutics shares fell by more than 12% on the news the US Food and Drug Administration (FDA) requires more time for its review of the company’s Biologics License Application (BLA) of its investigational gene therapy SRP-9001 for Duchenne muscular dystrophy.
The company said the FDA requires additional time to complete the review, including final label negotiations and postmarketing commitment discussions, and it expects to complete the review by June 22, 2023, almost a month later than its previous regulatory action date of May 29.
The FDA has indicated, subject to completion of the review, that it is working toward potentially granting an accelerated approval for SRP-9001, initially to be used in Duchenne patients aged between 4 and 5, Sarepta said.
The proposed confirmatory study is EMBARK, a Phase 3 trial of SRP-9001, which is fully enrolled with top-line results expected in the fourth quarter of 2023.
According to Sarepta, if the trial meets its objectives, the FDA intends to consider a non-age-restricted expansion of the SRP-9001 label based on its review of the EMBARK data.
The company noted it would remain in a quiet period for the duration of the BLA review.
Sarepta shares were down 12.4% at US$128.7 late on Wednesday morning.
Contact the author at emily.jarvie@proactiveinvestors.com
Follow her on Twitter @emilyjjarvie