MILAN, Italy — September 30, 2026
Italfarmaco S.p.A. presented new long-term data from the ongoing Phase 3 EPIDYS open-label extension study suggesting that givinostat (Duvyzat) may slow the decline of upper limb function in boys with Duchenne muscular dystrophy (DMD). The findings were presented at the 31st Annual International Congress of the World Muscle Society (WMS) and compared outcomes among givinostat-treated patients with modeled counterfactual disease trajectories derived from an external natural history study. In patients evaluable at 12 months, the mean change in Performance of Upper Limb 2.0 (PUL 2.0) score was −0.78 with givinostat versus a predicted −4.57, representing a difference of 3.79 points and nominal p<0.001. At 24 months, the observed mean change was −2.26 compared with a predicted −8.55, a difference of 6.29 points and nominal p<0.001.
Givinostat Analysis Focuses on Upper Limb Function
The exploratory analysis evaluated upper limb function using the PUL 2.0 assessment, an outcome measure that becomes increasingly relevant as DMD progresses and patients lose ambulation. A total of 115 patients were evaluable for the 12-month analysis, while 96 patients were evaluable at 24 months. The study compared observed changes in patients receiving givinostat with predicted outcomes generated through an external natural history-based model representing disease progression in the absence of treatment. At 12 months, the observed mean decline was substantially smaller than the model-predicted decline, while the difference was also observed at 24 months. Italfarmaco said the findings suggest a slower average decline in upper limb function among treated patients, although the analysis is exploratory and relies on modeled external comparator trajectories rather than a concurrent randomized control group.
EPIDYS Data Expand Long-Term Givinostat Evidence
The new findings add to the long-term clinical dataset being generated through the EPIDYS open-label extension study. The Phase 3 EPIDYS trial was designed as a randomized, placebo-controlled study evaluating the efficacy and safety of givinostat in patients with DMD, while the ongoing extension is intended to provide additional information on longer-term outcomes. The latest analysis extends evaluation beyond ambulatory measures to an aspect of disease progression that can influence independence and the ability to perform everyday activities. Because the upper limb findings were generated using counterfactual trajectories from a single-centre natural history study, the results should be interpreted in the context of potential limitations associated with external-model comparisons and should not be considered equivalent to evidence from a concurrent controlled trial.
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Givinostat Approved for DMD in US and EU
Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that regulates excessive HDAC activity associated with DMD muscle pathology. Its mechanism is designed to affect biological processes involved in muscle maintenance and repair and is independent of the specific dystrophin gene mutation causing DMD. Givinostat is approved for DMD in multiple regions, including the United States and European Union, for patients aged 6 years and older, with regional differences in ambulatory-status criteria. The latest WMS presentation provides additional information on upper limb function during long-term treatment, while further evaluation of the durability and clinical significance of these findings continues through the EPIDYS open-label extension.
Source :Italfarmaco, press release
