Italfarmaco's Duchenne drug gets FDA rare pediatric disease designation

The US Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Italfarmaco Group’s givinostat for the treatment of Duchenne muscular dystrophy (DMD), which allows it an expedited review process.

Italfarmaco has also announced the completion of patient enrollment in the EPIDYS Phase III trial, and remains on track with its proposed timelines for reporting top-line data in June 2022.

Paolo Bettica, chief medical officer at the Italian pharma company, said: “This is the third regulatory designation we have received from the FDA for givinostat after the Orphan Drug designation and Fast Track designation and reflects the agency’s recognition of givinostat’s potential to treat DMD, a devastating genetic disease.