May 29, 2025 — Earlier today, the U.S. Food and Drug Administration (FDA) decided against approval of elamipretide for use in patients impacted by the rare mitochondrial disease Barth syndrome. UMDF President and CEO Kristen Clifford issued the following statement:
“We are deeply disappointed in the FDA’s decision to not approve the current new drug application for elamipretide for the treatment of Barth syndrome. While we appreciate guidance that could eventually provide a pathway for accelerated approval, that’s little consolation for patients where even a delay of a few days matter. Time is simply not on our side. As we stated in our letter to the FDA Advisory Committee this past fall, every patient living with mitochondrial disease deserves access to therapies that are shown to safely enhance quality of life. We believe elamipretide met that standard for Barth syndrome and the advisory committee clearly agreed. We thank the Barth Syndrome Foundation for their leadership in this process and we look forward to working together to explore every option to fill this huge unmet medical need.”

Philip Yeske, PhD, UMDF Interim Managing Director and Science & Alliance Officer
and Emily Milligan, Executive Director of Barth Syndrome Foundation
at the FDA Advisory Committee Meeting to Review Elamipretide on Sept. 10