Dyne Therapeutics, Inc. Receives FDA Orphan Drug and Rare Pediatric Designations for Dyne-251 for the Treatment of Duchenne Muscular Dystrophy

Dyne Therapeutics, Inc. announced that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping, was granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations. DYNE-251 is being evaluated in the Phase 1/2 DELIVER clinical trial. Orphan drug designation is granted by the FDA to drugs or biological products intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the United States.

Under the FDA's rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a rare pediatric disease.