Long-Term Safety of Givinostat in Patients with Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

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Background/Purpose: Givinostat, an oral histone deacetylase inhibitor, has been investigated for Duchenne muscular dystrophy (DMD) treatment in patients aged ≥6 years. Safety data from the ongoing long-term study of givinostat were evaluated (NCT03373968). Methods: This is an ongoing open-label, long-term safety, tolerability, and efficacy study of givinostat in boys who completed or were screened but not randomized in previous DMD givinostat studies. Results: As of December 31, 2021, data cutoff, 194 patients have enrolled: givinostat ( n = 110), prior placebo ( n = 54), and not included in prior study ( n = 30). All patients received givinostat and corticosteroids. Safety data were evaluated for patients who received ≥1 givinostat administration. The mean duration of givinostat exposure was 616, 506, and 451 days in the givinostat, prior placebo, and not-included groups, respectively. Overall, 87.1% of patients reported ≥1 treatment-emergent adverse event (TEAE), with similar incidence among all groups. Most TEAEs were mild to moderate in severity. Among the most frequently reported TEAEs (≥10% of the overall population), diarrhea was reported more frequently by the prior placebo (27.8%) and not-included (36.7%) groups compared with the givinostat group (18.2%). More falls were reported by the givinostat group (20.0%) compared with the prior placebo (9.3%) and not-included (13.3%) groups. More reports of thrombocytopenia were observed in the prior placebo group (20.4%) compared with the givinostat (9.1%) and not-included (10.0%) groups. The givinostat group reported pyrexia (17.3%), increased blood triglycerides (12.7%), and decreased platelet count (9.1%); the incidence of these TEAEs in the prior placebo group was 13.0, 16.7, and 14.8%, respectively, and 6.7% for each TEAE in the not-included group. Decreased platelets and increased triglycerides followed by stabilization are consistent with givinostat treatment initiation. Conclusion: These results are consistent with the known safety profile of givinostat. Publication History Article published online: 26 September 2025 © 2025. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

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