Dyne Therapeutics’ DYNE-251 Receives FDA Breakthrough Therapy Designation
Dyne Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The decision is based on results from the ongoing global DELIVER trial.
The designation is intended to accelerate the development of medicines for serious conditions and provides Dyne with closer regulatory guidance, eligibility for Rolling Review and the possibility of a shortened eight-month review period.
DYNE-251 has also been awarded Fast Track, Orphan Drug and Rare Paediatric Disease designations in the United States and Orphan Drug designation in Europe.
The DELIVER trial is assessing DYNE-251 in 32 patients at a registrational dose of 20 mg/kg every four weeks, with data expected in late 2025.
Dyne plans to submit for Accelerated Approval in the U.S. in early 2026 and is preparing to pursue approval in other regions.
DMD is a rare genetic muscle-wasting disorder affecting around 12,000 people in the U.S. and 16,000 in the EU, with urgent demand for treatments that improve patient function.
