On Track to Report Initial Data from the Global, Multiple Ascending Dose Phase 1/2 ACHIEVE Clinical Trial in the Second Half of 2023
DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with myotonic dystrophy type 1 (DM1). (Credit: Christina Victoria Craft on Unsplash)
Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is being evaluated in the Phase 1/2 global ACHIEVE clinical trial with initial data on safety, tolerability and splicing from the multiple ascending dose, placebo-controlled portion of the trial anticipated in the second half of 2023.
“Receiving FDA orphan drug designation for DYNE-101 underscores the importance of bringing new treatment options to people living with DM1, a rare, devastating disease with no approved therapies. We believe DYNE-101 has the potential to be a transformative therapy which is why we designed the ACHIEVE trial to be registrational,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. “We recognize the sense of urgency within the DM1 community and look forward to sharing initial data from the ACHIEVE trial.”
Orphan drug designation is granted by the FDA to drugs or biologics 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 Orphan Drug Act, orphan drug designation qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.
Source: Company Press Release