US approved oral nonsteroidal therapy for Duchenne myodystrophy

The U.S. Food and Drug Administration (FDA) has approved the first orally administered low-molecular-weight non-steroidal drug for the treatment of Duchenne myodystrophy, the organization said in a press release. It was givinostat from the group of histone deacetylase inhibitors (HDACs), which reduces the expression of a number of cytokines and consequently reduces inflammation and loss of muscle mass. It was approved for use in patients aged six years and older with any genetic variant of the disease.

The FDA decision was prompted by the results of an 18-month double-blind, randomized, placebo-controlled Phase III trial in which givinostat was added to standard steroid therapy. At the end of the follow-up period, patients receiving the active drug performed significantly better on the four-step elevation test and the NSAA scale than those in the control group. Givinostat for the therapy of Duchenne myodystrophy was considered in priority order under accelerated procedure as an orphan drug for the treatment of rare pediatric diseases (RPD).