Scott Baver, PhD, Vice President of Medical Affairs at ITF Therapeutics, discusses long-term safety and efficacy data on givinostat for patients with Duchenne muscular dystrophy (DMD).
DMD is a rare neuromuscular disorder characterized by progressive muscle wasting. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive weakness and atrophy of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. Muscle weakness is usually noticeable in early childhood. DMD is caused by genetic changes in the DMD gene.
Recently, positive long-term safety and efficacy data from the phase 2 and phase 3 EPIDYS clinical trials of givinostat in patients with DMD was published in Annals of Clinical and Translational Neurology.
Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that regulates excessive HDAC activity that occurs in muscles affected by DMD. This helps restore expression of key genes and biological processes necessary for muscle maintenance and repair. Givinostat was approved by the U.S. Food and Drug Administration (FDA) in March 2024 for the treatment of patients with DMD ages six years and older.
The analysis from the EPIDYS trials included multiple cohorts of boys with DMD who received givinostat over various durations. Using propensity matching methods, 142 patients receiving givinostat and 142 from the natural history cohort were included in the analyses. Results indicated that long-term treatment with givinostat, in combination with corticosteroids, delayed disease progression in ambulant patients ages six years and older with DMD.
Clinical benefit was also observed in key mobility functions such as ability to rise from floor, where givinostat may delay loss of ability by a median of 2 years. Loss of ability to climb four stairs was delayed by 3.3 years and loss of ability to walk was delayed by 2.9 years. Additionally, givinostat was generally well-tolerated across all cohorts and no new safety signals emerged during long-term treatment.
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To learn more about DMD and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/