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Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy (DMD)
- Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in early 2026 - WALTHAM, Mass., Aug. 04, 2025 (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 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. “This Breakthrough Therapy Designation for DYNE-251 is a testament to its potential as a next-generation therapy designed to bring meaningful functional improvement to individuals with DMD for whom exon 51 skipping can lead to the production of near full-length dystrophin,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “As we’ve previously disclosed, DYNE-251 has demonstrated sustained functional improvement through eighteen months, as assessed by key measures such as time to rise and stride velocity 95th centile. The level of near-full-length dystrophin expression observed marks a significant step forward to potentially deliver meaningful benefits to patients.” Both of Dyne’s lead programs now have FDA Breakthrough Therapy Designation, with DYNE-101 having been granted Breakthrough Therapy Designation in myotonic dystrophy type 1 (DM1) earlier this year. The FDA grants Breakthrough Therapy Designation to expedite the development and review of drugs that are intended to treat a serious condition with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints. This designation offers benefits to DYNE-251 in the U.S. including: Enhanced FDA support, including senior-level involvement to guide efficient development as well as decision-making Early and frequent communication with FDA reviewers on trial design and regulatory strategy Rolling and Priority Review eligibility, potentially reducing the Biologics License Application (BLA) review timeline from 12 to 8 months DYNE-251 in DMD DYNE-251 has been granted Fast Track, Orphan Drug a...