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EC Grants Orphan Drug Designation For Dyne Therapeutics' DYNE-251 For Treatment Of Duchenne Muscular Dystrophy

Benzinga·04/24/2025 11:32:54
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- Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months -

- Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 -

WALTHAM, Mass., April 24, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD who are amenable to exon 51 skipping. Long-term clinical data from the ongoing DELIVER trial of DYNE-251 that demonstrated unprecedented and sustained functional improvement at the selected registrational dose were presented in March at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. Functional assessments in the DELIVER trial include Stride Velocity 95th Centile (SV95C), an objective digital outcome that is accepted as a primary endpoint for DMD clinical trials in Europe.