March 3, 2020: Stealth BioTherapeutics announced that the U.S. FDA has granted Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition. Under the RPD classification system of the FDA, the FDA may issue a priority review voucher to a sponsor who receives approval for a “rare pediatric disease,” a severe and life-threatening disease that mainly affects persons aged 18 years and under 200,000 in the United States.
Stealth BioTherapeutics is eligible for a voucher following FDA approval of elamipretide for Barth syndrome, which can be used to receive priority consideration for a subsequent human drug application in complying with the applicable statutory requirements associated with the RPD programme.
Barth syndrome is an ultra-rare genetic condition characterized by cardiac defects that often result in heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed development. Barth syndrome occurs nearly exclusively in males and is estimated to affect one in 200,000 to 400,000 people around the world. There are no FDA- or EMA-approved treatments for Barth syndrome patients at this time.
https://www.streetinsider.com/PRNewswire/FDA+Grants+Rare+Pediatric+Disease+Designation+to+Stealth+BioTherapeutics+for+Elamipretide+for+the+Treatment+of+Barth+Syndrome/16561448.html
