Investing.com -- Shares of Dyne Therapeutics (NASDAQ: DYN ) climbed 3.9% following the announcement that the European Commission has granted orphan drug designation to the company’s lead product candidate, DYNE-251, for the treatment of Duchenne muscular dystrophy (DMD). This regulatory milestone is recognized as a significant step for the biotech firm, which specializes in developing therapies for genetically driven neuromuscular diseases.
The orphan drug designation is particularly noteworthy as it can provide Dyne with a range of benefits, including reduced regulatory fees, assistance with clinical protocols, eligibility for research grants, and up to a decade of market exclusivity in the European Union upon approval. This status is granted to treatments for rare diseases that affect fewer than five in 10,000 people within the EU.
DYNE-251 is currently being assessed in the Phase 1/2 DELIVER global clinical trial, which targets individuals with DMD amenable to exon 51 skipping. Recent long-term clinical data from this trial, presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in March, showed sustained and unprecedented functional improvement at the selected registrational dose. The trial’s functional assessments, including Stride Velocity 95th Centile (SV95C), have been accepted as a primary endpoint for DMD clinical trials in Europe.
Dr. Doug Kerr, the Chief Medical Officer of Dyne, expressed optimism about the trial’s results and the orphan drug designation, stating, "We are pleased that the EC has granted orphan drug designation to DYNE-251, reinforcing our belief that our next-generation exon 51 skipping investigational therapy for DMD may be able to bring clinically meaningful functional improvement to those living with this devastating disease."
With full enrollment of the registrational expansion cohort in the DELIVER trial complete, Dyne Therapeutics anticipates sharing further data from this cohort in late 2025 and aims to proceed with its first regulatory submissions in early 2026. The company also received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) in March 2023, marking a significant period of regulatory achievements that could potentially enhance the company’s prospects and investor confidence.
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