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
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
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.
REFERENCES
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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