A significant advance in research on therapy for Duchnne Muscular Dystrophy: FDA approves Givinostat after the results of the Epydis study

The Food and Drug Administration (FDA – USA) has approved Givinostat for the treatment of Duchenne Muscular Dystrophy (DMD), thanks to the results obtained from the phase 3 EPIDYS clinical trial.

The drug Givinostat is a histone deacetylase inhibitor developed by Italfarmaco, Italy, for the treatment of DMD. From the early stages of development, the neurologists and researchers of the Dino Ferrari Center, Department of Medical, Surgical Pathophysiology and Transplant, University of Milan and Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, directed by Prof. Giacomo Comi, collaborated on the clinical studies that led to this first significant result.

In a study just published in Lancet Neurology, treatment with givinostat showed a significant reduction in the decline in motor skills in boys with DMD.

The multicenter, randomized, double-blind, placebo-controlled phase III trial, conducted by Italafarmaco, involved 179 participants in 11 countries, demonstrating that givinostat effectively slows the deterioration of motor function compared to placebo. “These results represent concrete hope for patients and their families,” said Prof. Comi. “DMD is a condition that primarily affects children, leading to a progressive loss of muscle strength. Our study highlights the potential of givinostat to modify the course of this devastating disease.”

The EPIDYS trial is the result of a large international collaboration carried out by Italfarmaco in difficult circumstances during the COVID-19 pandemic and of a ten-year commitment to research on DMD. Prof. Comi underlined the importance of international cooperation and continuous support for scientific research to combat neuromuscular diseases.

For further information on the approval of the drug by the FDA, please consult this website.

For the results of the study and future research directions, please consult the publication in the Lancet Neurology.