Sarepta Therapeutics to seek FDA accelerated approval for SRP-9001 in DMD


Sarepta Therapeutics said that it plans to file a biologics license application (BLA) with the US Food and Drug Administration (FDA) to seek accelerated approval for SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD).

The investigational DMD gene therapy is being co-developed with Roche under a $2.85bn deal signed in 2019. Sarepta Therapeutics is looking to submit a BLA for the gene therapy candidate this fall.

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The decision to pursue SRP-9001 accelerated approval comes after feedback from the FDA and a thorough and in-depth review by the company.

Doug Ingram — Sarepta Therapeutics president and CEO said: “We look forward to a collaborative review commencing this year and running through the first half of 2023.

“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.”

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SRP-9001 has FDA fast track designation, which was granted in July 2020. The DMD gene therapy candidate also has a rare pediatric disease (RPD) designation in the US apart from orphan drug status in the US, Switzerland, the European Union, and Japan.

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