Today, Italfarmaco’s U.S. affiliate, ITF Therapeutics LLC, announced seven abstracts for the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Dallas, Texas, March 16-19, 2025. Data from the Phase 3 EPIDYS study and the company’s open-label extension study in DMD patients treated with givinostat are shown on posters. Disease progression and respiratory function data are used to evaluate givinostat’s long-term safety and efficacy. [What is Duvyzat (Givinostat)?]
One poster presented by Krista Vandenborne, Ph.D., Chair, Department of Physical Therapy, University of Florida, includes data from the Phase 3 EPIDYS study and from an ongoing open-label extension study evaluating the long-term safety, tolerability, and efficacy of givinostat in boys aged ≥ 6 years with DMD.
Using propensity score matching based on baseline functional test results and type of steroid, 142 patients from EPIDYS and the extension study treated with givinostat and steroids were matched with 142 patients from two DMD natural history studies treated with steroids only. Results were assessed based on the median age at which DMD progression milestones such as persistent loss to perform 4-stair climb, loss of rise from floor, and loss of ambulation, occurred. The median age at loss of ambulation in patients treated with givinostat was 18.1 years, compared to 15.2 years in the control group.
