Stresa, Italy – ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, announced new data on givinostat (DUVYZAT®) for Duchenne muscular dystrophy (DMD) at the 2025 Neuromuscular Study Group (NMSG) Annual Scientific Meeting.
The presentations highlight insights from the pivotal Phase 3 EPIDYS trial and related studies, including flexible dosing, safety, biomarker correlations, and respiratory outcomes.
Key Data Highlights
- Weight-Based Flexible Dosing (Poster #1213): Analysis of dose adjustments in EPIDYS showed impact on 4-stair climb (4SC) time and North Star Ambulatory Assessment (NSAA) scores at week 72.
- Thrombocytopenia Characterization (Poster #1207): Expanded analysis of platelet count changes confirmed thrombocytopenia as a manageable, dose-related adverse event.
- Vastus Lateralis Fat Fraction (Poster #1211): VLFF changes correlated with functional outcomes, reinforcing its role as a potential biomarker for disease progression.
- Respiratory Function (Poster #1208): Data from EPIDYS and extension studies suggest slower decline in forced vital capacity (FVC) compared with natural history controls.
Expert Perspectives
“These presentations reflect our focus on supporting clinicians with data that clarify the benefit-risk profile of givinostat, including flexible dosing strategies,”
- Scott Baver, Ph.D., VP, Head of Medical Affairs, ITF Therapeutics.
“Together, these findings deepen our understanding of givinostat’s use in DMD and may help guide clinical decision-making,”
- Dr. Aravindhan Veerapandiyan, University of Arkansas for Medical Sciences.
About DUVYZAT® (givinostat)
- Class: Oral histone deacetylase (HDAC) inhibitor
- Indication: Approved for DMD in patients ≥6 years
- Mechanism: Regulates excessive HDAC activity in dystrophic muscle, helping restore gene expression and muscle repair processes independent of dystrophin mutation type.
Safety Profile
- Common adverse reactions (>5%): Diarrhea, abdominal pain, thrombocytopenia, nausea/vomiting, hypertriglyceridemia, pyrexia, myalgia, rash, arthralgia, fatigue, constipation, decreased appetite.
- Warnings/Precautions: Monitor for hematologic changes, triglyceride elevations, gastrointestinal disturbances, and QTc prolongation. Regular ECG and blood count monitoring recommended.
About ITF Therapeutics and Italfarmaco
- ITF Therapeutics LLC (launched 2024) is focused on rare diseases, with programs built in collaboration with advocacy and research communities.
- Italfarmaco (founded 1938, Milan) is a private global pharma company with operations in 90+ countries, active in neurology, rare diseases, immuno-oncology, and cardiovascular care.
