The USFDA has recently approved givinostat, a nonsteroidal oral suspension, for treating Duchenne muscular dystrophy (DMD) in patients aged six years and above. Givinostat is a histone deacetylase inhibitor. The molecular mechanism behind the effect of givinostat on DMD is not yet elucidated. Clinical evidence The efficacy of givinostat was evaluated in a phase 3 clinical trial involving 179 DMD patients on corticosteroids throughout the study.
Patients treated with givinostat displayed significantly less decline in the time to climb four stairs. In the 4-stair climb test, the mean change of time in climbing stairs from baseline to month 18 was 1.25 seconds among patients treated with givinostat compared to 3.03 seconds among patients who received a placebo. In addition, givinostat-treated patients showed less worsening of motor function in the North Star Ambulatory Assessment…
