Dyne Therapeutics Inc (NASDAQ:DYN) stock climbed 3.6% following the announcement that the U.S. Food and Drug Administration (FDA) awarded Breakthrough Therapy Designation to its DYNE-251 treatment targeting Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.
This designation was granted based on promising data from Dyne’s ongoing DELIVER clinical trial. The breakthrough status offers enhanced FDA support, including senior-level involvement, earlier and more frequent communication with reviewers, as well as eligibility for rolling and priority review — potentially shortening the review period from 12 months to 8 months.
“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, chief medical officer of Dyne.
The company noted that DYNE-251 has demonstrated sustained functional improvements through 18 months, evaluated by key metrics such as time to rise and stride velocity 95th centile.
This marks the second breakthrough therapy designation for Dyne’s lead candidates, with DYNE-101 receiving similar recognition earlier this year for myotonic dystrophy type 1.
Dyne has completed enrollment of 32 patients in the Registrational Expansion Cohort of the DELIVER trial, with data expected in late 2025. The company anticipates submitting a Biologics License Application for U.S. Accelerated Approval in early 2026.
DYNE-251 also holds FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations, along with Orphan Drug status from the European Medicines Agency for DMD treatment.
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