Italfarmaco reports new MRI and long-term extension data from the EPIDYS trial and OLE study, showing givinostat (Duvyzat®) preserves muscle tissue, reduces fat replacement, and extends ambulation in Duchenne muscular dystrophy, with consistent long-term safety.
Written By: Shaik Yasmeen, PharmD
Reviewed By: Pharmacally Editorial Team
Italfarmaco has presented new clinical data supporting the long-term benefits of givinostat (Duvyzat®) in Duchenne muscular dystrophy (DMD) during the 19th International Congress on Neuromuscular Diseases (ICNMD) 2026 in Florence, Italy. The findings include new quantitative magnetic resonance imaging (MRI) analyses from the pivotal Phase 3 EPIDYS trial and updated results from its ongoing open-label extension (OLE) study, providing further evidence that the therapy preserves muscle tissue, limits fat replacement, and maintains a favorable long-term safety profile.
The MRI analyses demonstrated statistically significant improvements in muscle composition among patients treated with givinostat compared with placebo. Across selected lower limb muscles, treatment increased contractile cross-sectional area (cCSA), an indicator of preserved functional muscle tissue, while reducing fat fraction, a marker of progressive muscle degeneration. These imaging findings provide objective evidence that the treatment slows structural changes associated with disease progression.
Mutation-Independent Approach to Slowing Disease Progression
Duchenne muscular dystrophy is a rare, progressive genetic disorder caused by mutations in the dystrophin gene, leading to continuous muscle damage, declining motor function, and eventual loss of ambulation. Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that targets excessive HDAC activity observed in DMD muscle. By restoring the expression of genes involved in muscle repair and maintenance, the therapy works independently of the underlying dystrophin mutation, making it applicable across a broad DMD population.
Phase 3 MRI Findings and Long-Term Extension Outcomes
The Phase 3 EPIDYS trial (NCT02851797) compared givinostat with placebo in patients with DMD, with MRI-based assessments included as exploratory measures of disease progression. Patients receiving givinostat showed improvements in cCSA ranging from 0.43 cm² to 1.20 cm² compared with placebo, while fat fraction was reduced by 3.4% to 4.6% across evaluated lower limb muscles.
Updated interim data from the ongoing OLE study included 225 patients receiving long-term treatment. Investigators reported a median age at persistent loss of ambulation of 17.3 years (95% CI: 15.5–18.1 years), substantially later than the 11.0 to 13.4 years previously reported in published natural history studies of corticosteroid-treated patients. Although these comparisons were made against historical datasets rather than a concurrent control group, they contribute to the growing understanding of long-term clinical outcomes with givinostat.
Safety findings remained consistent throughout extended follow-up. Patients treated with givinostat for more than 10 years in the clinical trial setting showed no new safety signals, supporting the drug’s established safety profile.
Company Highlights Clinical Significance
Commenting on the findings, Scott Baver, Ph.D., Vice President and Head of Global Medical Affairs, Rare Diseases at Italfarmaco, said the MRI analyses and long-term observational data add to the growing body of evidence for givinostat by providing deeper insight into disease progression and long-term treatment outcomes in patients with DMD.
Ongoing Follow-Up to Define Long-Term Benefit
Givinostat is currently approved for the treatment of Duchenne muscular dystrophy in multiple regions, including the United States, European Union, United Kingdom, and United Arab Emirates, for patients aged six years and older. Labeling differs by region, particularly regarding ambulatory status. The ongoing extension study continues to evaluate long-term efficacy and safety, with additional follow-up expected to further define the therapy’s role in slowing disease progression.
What This Means for Patients
The findings suggest that givinostat may help patients with Duchenne muscular dystrophy remain physically active for longer by slowing the loss of healthy muscle and reducing the replacement of muscle with fat, a hallmark of disease progression. In the long-term extension study, patients receiving givinostat remained ambulatory for a median of 17.3 years, compared with 11 to 13.4 years reported in historical studies of corticosteroid-treated patients. While this comparison was not made against a concurrent control group, it indicates the potential for delaying one of the most significant milestones in DMD.
For patients and families, preserving muscle function for longer could translate into greater independence in everyday activities, delayed need for wheelchair use, and an extended period of mobility. The absence of new safety concerns after more than 10 years of treatment also provides reassurance about the therapy’s long-term use.
Reference
Presentation of New Clinical Data on Givinostat in Duchenne Muscular Dystrophy
About the Writer
Shaik Yasmeen (LinkedIn) is a Pharm.D graduate with interests in clinical pharmacy, pharmacovigilance, and medical writing. She has gained experience through hospital clinical postings, patient case reviews, case presentations, and literature evaluation. Passionate about evidence-based healthcare, she is committed to creating accurate and engaging medical content while continuously expanding her professional knowledge.