The U.S. Food and Drug Administration (FDA) has approved givinostat for the treatment of patients with all genetic variants of Duchenne muscular dystrophy (DMD).
DMD is caused by genetic changes in the DMD gene that leads to a reduction in functioning levels of the protein dystrophin. The disease largely affects the muscles, leading to progressive muscle wasting. The symptoms include progressive weakness and loss of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. Diagnosis of DMD is based on symptoms, clinical exam, and the result of a biopsy. The result of genetic testing may also help confirm the diagnosis.
Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that targets pathogenic processes, reducing inflammation and muscle loss.
In the video clip below, Paolo Bettica, MD, PhD, Vice President of Research and Development at ITF Therapeutics, discusses the mechanism of action of givinostat to treat DMD.
The efficacy of givinostat was tested through a randomized, double-blind, placebo controlled, 18-month, phase 3 study. The study achieved its primary endpoint of significant decline in time it took to complete the four-stair climb test (measuring muscle function) following the 18-month period. The secondary endpoint, less worsening of North Star Ambulatory Assessment (NSAA) score, was also observed.
In the clip below, Dr. Bettica provides more details on the study and the results.
For more information on DMD, and other rare musculoskeletal disorders, visit https://checkrare.com/diseases/musculoskeletal-diseases/