Dyne Therapeutics submits FDA application for DMD treatment

Dyne Therapeutics Inc. (NASDAQ: DYN) submitted a Biologics License Application to the U.S. Food and Drug Administration for z-rostudirsen, a treatment for Duchenne muscular dystrophy patients amenable to exon 51 skipping.

The Waltham, Massachusetts-based company filed for accelerated approval based on dystrophin as a surrogate endpoint. The proposed dosing regimen is 20 mg/kg administered intravenously once every four weeks.

The application is based on results from the registrational expansion cohort of the DELIVER trial, where treatment with z-rostudirsen resulted in increased dystrophin production with functional improvement observed across multiple clinical endpoints, according to the company's press release.

"This is a significant milestone for both our company and the DMD community," said John Cox, president and chief executive officer of Dyne. "Despite the availability of approved therapies, there remains a significant unmet need in DMD for treatments with compelling efficacy, a favorable safety profile and improved dosing convenience."

Dyne requested Priority Review for the application, which would shorten the review process from 10 months to six months following the FDA's 60-day filing review period. The company expects a potential U.S. launch in the first quarter of 2027, assuming Priority Review is granted and approval is received on the anticipated timeline.

Z-rostudirsen has received Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug designation from the FDA, European Medicines Agency and Japan's Ministry of Health, Labour and Welfare.

The company is also developing four additional candidates for DMD treatment targeting different exons: DYNE-253, DYNE-245, DYNE-244 and DYNE-255 for exons 53, 45, 44, and 55, respectively.

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