Dyne Therapeutics 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.
DYNE-251: Breakthrough Therapy Designation
“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.”
Learn More: Upcoming Exon 51 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
