Sarepta to seek fast review of its BLA for gene therapy SRP-9001 to treat DMD

US precision genetic medicine for rare diseases developer Sarepta Therapeutics (Nasdaq: SRPT) said on Friday that it intends to submit a Biologics License Application (BLA) seeking accelerated approval for SRP-9001 (delandistrogene moxeparvovec) to treat ambulant individuals with Duchenne muscular dystrophy.

SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Swiss pharm giant Roche (ROG: SIX). The news pushed Sarepta’s shares up 8% to $93.44 by close of trading on Friday.

“We are delighted to confirm that based on FDA feedback received following a thorough and in-depth review, we intend to submit a BLA for our SRP-9001 gene therapy to treat Duchenne muscular dystrophy this fall. We look forward to a collaborative review commencing this year and running through the first half of 2023,” said Doug Ingram, president and chief executive of Sarepta Therapeutics. “Duchenne robs children daily and hourly of their muscle, stealing them bit by bit from their families and loved ones. Guided by rigorous science and productive regulatory discussions, our goal is to move with the urgency desperately needed by the patient community, and our upcoming BLA filing for SRP-9001 serves that goal,” he noted.