After a full day of testimony that consistently demonstrated the need and efficacy of elamipretide for the treatment of Barth Syndrome, an FDA advisory committee correctly recommended approval of the therapy’s new drug application. The committee’s decision reinforces our belief that, when it comes to families experiencing mitochondrial disease, patients deserve access to any therapy proven to safely improve quality of life for these devastating diseases. We thank the hard work of Stealth BioTherapeutics, Barth Syndrome Foundation, and many families, clinicians, and researchers who fought to get us here. And while the final decision on elamipretide approval now sits with the FDA, we have confidence that once all evidence is reviewed, they will come to the same conclusion. There is simply too much on the line for our community to come to any other conclusion.
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