Evaluating Givinostat's Efficacy and Safety in Duchenne Muscular Dystrophy: Marcello Paglione, PhD


The executive medical lead at Italfarmaco SpA talked about data from both animal models and clinical trials supporting the efficacity of givinostat in patients with Duchenne muscular dystrophy at MDA 2024. [WATCH TIME: 5 minutes]

WATCH TIME: 5 minutes

"In the post hoc analysis, the results are very much consistent with the prespecified analysis and confirmed what we have seen. I want to conclude saying that the results of the large phase 3 trial support our application for the approval.”

In March 2024, the FDA approved Italfarmaco SpA's givinostat (Duvyzat), a proprietary histone deacetylase (HDAC) inhibitor, as a treatment for patients with Duchenne muscular dystrophy (DMD).1 The treatment, the first nonsteroidal drug approved for all genetic variants of DMD, is designed to inhibit HDACs, enzymes that prevent gene translation, by changing the 3-dimensional folding of DNA in the cell. The supporting data for the approval of givinostat comes from the phase 3 EPIDYS trial (NCT02851797).

EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Of these, 120 boys formed the target population. In a new supportive post hoc analysis from EPIDYS, findings showed a positive significant difference with givinostat in motor function among patients with DMD in comparison with controls. Presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 3-6, in Orlando, Florida, these results emphasize the efficacy of givinostat in a broader context.2

Marcello Paglione, PhD, executive medical lead at Italfarmaco SpA, sat down with NeurologyLive® at the conference to discuss the key findings regarding givinostat’s mechanism of action in DMD as observed in the research presented at MDA 2024. He spoke about the results from animal models compared with those from clinical trials. In addition, Paglione talked about the most common adverse events associated with givinostat in the phase 3 trial, and how they were managed among the participants.

1. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. FDA. News release. March 21, 2024. Accessed April 2, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
2. Mercuri EM, Brogna C, Zaidman C, et al. The Epidys Givinostat Study in DMD: supportive results. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Poster M163.
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