Meeting Scheduled with Cardiovascular and Renal Drugs Advisory Committee on October 10, 2024 NEEDHAM, Mass. , Sept. 9, 2024 /PRNewswire/ -- Stealth BioTherapeutics Inc.
(the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced today that the U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting with the Cardiovascular and Renal Drugs Advisory Committee (CRDAC) on October 10, 2024 to evaluate the New Drug Application (NDA) for elamipretide, the first potential therapy for Barth syndrome.
This ultra-rare, progressive disease affects approximately 150 individuals in the United States , causing severe cardiac and skeletal muscle manifestations that significantly reduce life expectancy. The NDA has been granted priority review, with a Prescription Drug User Fee Act (PDUFA) action date of January 29, 2025 . "We are grateful to the patients, families, and physicians who participated in the studies that supported this new drug application, as well as to the patient and medical community members who requested a review of elamipretide as the first potential therapy for Barth syndrome," said Reenie McCarthy , Chief Executive Officer.
"We look forward to meeting with the Cardiovascular and Renal Drugs Advisory Committee to discuss elamipretide's potential to improve the lives of individuals living with Barth syndro.