FDA misses PDUFA goal for Stealth’s Barth syndrome treatment

FDA has missed another PDUFA review deadline, this time for a treatment that a small biotech is developing for an ultrarare disease.

Stealth Biotherapeutics Inc. announced Tuesday that FDA has not provided a reason for its inability to meet the April 29 PDUFA goal for elamipretide to treat for Barth syndrome. The progressive, life-threatening condition affects approximately 130 people in the U.S. and about 300 globally, and currently has no approved therapies. ...