FDA Grants Priority Review to Sarepta's DMD Gene Therapy
The FDA has accepted Sarepta's Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne Muscular Dystrophy (DMD).
The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. Sarepta is responsible for SRP-9001's U.S. application. The company is working with Roche for commercialization outside the country.
Studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment.
"The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne," Tracy Sorrentino, executive director of corporate affairs, told BioSpace. "Within the context of Duchenne and other rare diseases, it's a very robust sample size and one that will grow with data from EMBARK."
SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues.
Sarepta and Roche entered into a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001.
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