Italfarmaco Gains Rare Pediatric Disease Designation for Drug Treating Duchenne Muscular Dystrophy

The FDA has granted Italfarmaco a Rare Pediatric Disease designation for Givinostat, its investigational treatment for young males suffering from Duchenne Muscular Dystrophy.

In April, Italfarmaco disclosed positive interim study data indicating a slower disease progression in young males treated with Givinostat and steroids.  The company said it has now completed enrollment of 179 participants for a phase 3 trial and expects to release first results in June 2022.

Givinostat previously received Orphan Drug and Fast-Track designations from the FDA.