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Dyne Therapeutics: FDA Orphan Drug and Rare Pediatric Designations for DYNE-251

March 27, 2023

Defeat Duchenne Canada is pleased to announce that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy 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, including a Canadian clinical trial site in London, Ontario.

Dyne anticipates data from the Global Multiple Ascending Dose Phase 1/2 DELIVER Clinical Trial in the second half of 2023.

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 product approval for a rare pediatric disease.