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Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)
Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Submission for Accelerated Approval based on dystrophin as a surrogate endpoint -- In the registrational expansion cohort of the DELIVER trial, treatment with z-rostudirsen resulted in a robust and statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favorable safety profile1 -- Proposed dosing regimen of 20 mg/kg administered intravenously once every 4 weeks (Q4W) - WALTHAM, Mass., May 26, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) 20 mg/kg Q4W for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. “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. Z-rostudirsen was developed with the goal of delivering functional improvement with lower treatment burden for those living with this progressive disease. We look forward to continued engagement with the FDA to help facilitate a timely review, as we work toward bringing this potential best-in-class therapy to those in need as quickly as possible.” Dyne has requested Priority Review for the BLA, which, if granted, would shorten the review process from 10 months to 6 months following the FDA’s 60-day filing review period. Dyne continues to expect a potential U.S. launch of z-rostudirsen in Q1 2027, assuming the FDA grants Priority Review and approval is received on the anticipated timeline. In addition to z-rostudirsen, Dyne is advancing four development candidates (DYNE-253, DYNE-245, DYNE-244 and DYNE-255) for the potential treatment of DMD amenable to skipping of exons 53, 45, 44, and 55, respectively.About Zeleciment Rostudirsen (z-rostudirsen, also known as DYNE-251)Z-rostudirsen is an investigational therapeutic for individuals with...