Dyne Therapeutics receives EMA orphan drug designation for DYNE-251

Dyne Therapeutics (DYN) announced that the European Commission has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy. 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 Clinical & Scientific Conference. Functional assessments in the DELIVER trial include Stride Velocity 95th Centile, an objective digital outcome that is accepted as a primary endpoint for DMD clinical trials in Europe.