Exploring Long-Term Safety and Real-World Use of Givinostat in Duchenne Muscular Dystrophy: John F. Brandsema, MD

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"We’re all enthusiastic in the Duchenne community that we have more and more options to consider for treatment of the disease. We are always more hopeful about medications that have proven efficacy in trial, like givinostat."

Givinostat (Duvyzat; Italfarmaco SpA), an oral histone deacetylase inhibitor approved by the FDA for the treatment of Duchenne muscular dystrophy (DMD), had its safety and efficacy evaluated in the previously concluded phase 3 EPIDYS trial (NCT02851797).¹ The study’s open-label extension (OLE; NCT03373968), which remains ongoing, further assesses the long-term safety, tolerability, and efficacy of givinostat in patients who either completed or were screened but not randomized in prior studies. An interim analysis, presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 9-11, in Orlando, Florida, by lead author John F. Brandsema, MD, aimed to characterize safety outcomes observed to date in the OLE population.²

As of December 31, 2023, a total of 207 patients were enrolled, including those previously treated with givinostat (n = 119), those who had received placebo (delayed givinostat; n = 58), and treatment-naïve patients (n = 30). All participants received weight-based givinostat in combination with corticosteroids, with mean exposure exceeding 1000 days across groups. Nearly all patients (98.1%) experienced at least 1 treatment-emergent adverse event (AE), the majority of which were mild or moderate in severity. Common AEs included gastrointestinal symptoms, falls, thrombocytopenia, and elevated triglycerides, with some variation across groups. Overall, findings were consistent with the established safety profile of givinostat, with no new safety signals identified.

At the 2026 MDA Conference, Brandsema, a child neurologist at Children’s Hospital of Philadelphia, spoke with NeurologyLive^® to emphasize the importance of interpreting long-term safety data alongside real-world clinical experience. He noted that although givinostat has demonstrated efficacy and can be used in combination with other treatments, clinicians should remain attentive to tolerability considerations. Available evidence suggests that most AEs are manageable and may be addressed with dose adjustments without compromising treatment effect. However, he highlighted the need for continued investigation into potential late-onset laboratory abnormalities, cardiac safety, and outcomes in broader patient population.

Click here for more MDA 2026 coverage.

REFERENCES
1. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. FDA. News release. March 21, 2024. Accessed March 18, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
2. Brandsema J, Acsadi G, Alessi F, et al. Long-Term Safety of Givinostat in Patients With Duchenne Muscular Dystrophy: Interim Results From an Open-Label Extension Study. Presented at: MDA Clinical & Scientific Conference; March 8-11, 2026; Orlando, Florida. Abstract 51.