The FDA has communicated to Stealth BioTherapeutics that it would not meet elamipretide’s PDUFA target date for the treatment of Barth syndrome.

Barth syndrome is an ultra-rare genetic mitochondrial disease leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth.

Barth syndrome is an ultra-rare, life threatening pediatric mitochondrial disease which is known to affect less than 150 individuals in the United States and less than 300 individuals worldwide.