- Dyne Therapeutics (DYN, Financial) receives EU orphan drug designation for DYNE-251, a treatment for Duchenne Muscular Dystrophy (DMD).
- The designation offers reduced regulatory fees and potential 10-year market exclusivity upon approval.
- Data from a fully enrolled cohort in the Phase 1/2 DELIVER trial is expected in late 2025, with plans for a U.S. Biologics License Application (BLA) in early 2026.
Dyne Therapeutics, Inc. (DYN), a clinical-stage biotechnology company, announced that its investigational therapy DYNE-251 has been granted orphan drug designation by the European Commission for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is specifically targeted for DMD patients amenable to exon 51 skipping and is currently being evaluated in the Phase 1/2 DELIVER clinical trial.
The EMA's orphan drug designation for DYNE-251 follows promising clinical results showing sustained functional improvement over 18 months, as presented in March 2025. This designation confers several benefits, including reduced regulatory fees, clinical protocol assistance, and the potential for up to 10 years of market exclusivity in the European Union, contingent upon successful approval. This complements existing U.S. FDA orphan drug and rare pediatric disease designations awarded in March 2023.
Dyne has completed the enrollment of 32 patients in the registrational expansion cohort of the DELIVER trial, with results anticipated by the end of 2025. The company plans to submit a Biologics License Application (BLA) for accelerated U.S. approval in early 2026. DYNE-251's continued progress represents a significant step forward in addressing the needs of patients with this rare and progressive genetic disorder.
