John F. Brandsema, MD, on the Real-World Use of Givinostat in DMD

This interview was originally published on our sister site, NeurologyLive®.

"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 granted FDA approval for Duchenne muscular dystrophy (DMD), was the subject of a safety and efficacy evaluation in the now-completed phase 3 EPIDYS trial (NCT02851797).¹ The trial's open-label extension (OLE; NCT03373968), which is still underway, continues to assess the long-term safety, tolerability, and efficacy of givinostat among patients who either finished or were screened but not randomized in previous 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, a child neurologist at Children’s Hospital of Philadelphia, sought to characterize the safety outcomes observed thus far in the OLE population.²

Through December 31, 2023, a total of 207 patients had been enrolled — among them those previously treated with givinostat (n = 119), those who had received placebo (delayed givinostat; n = 58), and treatment-naïve patients (n = 30). All participants were given weight-based givinostat alongside corticosteroids, with mean exposure surpassing 1,000 days across all groups. Almost all patients (98.1%) experienced at least 1 treatment-emergent adverse event (AE), most of which were mild to moderate in severity. Frequently reported AEs included gastrointestinal symptoms, falls, thrombocytopenia, and elevated triglycerides, with some variation noted between groups. Taken together, these findings aligned with givinostat's known safety profile, and no new safety signals were identified.

At the conference, Brandsema spoke with CGTLive®’s sister site 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.