Statement from Barth Syndrome Foundation on FDA Advisory Committee Meeting
BOSTON (October 10, 2024) – The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to Barth syndrome and saving lives around the world through education, advances in treatments and finding a cure, today issued the following statement in response to the U.S. Food and Drug Administration (FDA) Cardiovascular and Renal Drugs Advisory Committee’s consensus to recommend approval of the New Drug Application (NDA) for elamipretide to treat Barth syndrome.
The 10-6 vote to recommend approval signals the advisory committee panel's assessment on elamipretide’s safety and effectiveness with its proposed intended use in Barth syndrome. The committee offers valuable perspective and non-binding recommendations for the FDA to factor in alongside other considerations during approval decisions. The FDA is expected to decide whether to approve the new drug application in January.
“We are deeply encouraged by today's positive recommendation from the FDA advisory committee for elamipretide’s approval,” said BSF Executive Director Emily Milligan, MPH.
“This milestone marks a major victory in our decade-long work to secure the first-ever FDA- approved treatment for Barth syndrome, a life-threatening disease. The lives of those with Barth syndrome depend on it. We strongly urge the FDA to move swiftly toward approving elamipretide for use in Barth syndrome, bringing patients closer to having sustained access to this critical therapy. Our plight is not just about Barth syndrome—it sets an important precedent for how we address the unmet needs of patients and families affected by many rare diseases.”
Barth syndrome is an ultra-rare, life-threatening genetic disorder primarily affecting males. There are no FDA-approved treatments and no other therapies in late-stage clinical development. Studies show elamipretide can boost muscle strength by 45% and heart function by 40%. Half of all patients from the original trial continue to take and benefit from this therapy almost seven years after initiating treatment, and for many, it is their only hope for survival.
Patients and families affected by Barth syndrome and leading advocates from the United Mitochondrial Disease Foundation, MitoAction, Friedreich’s Ataxia Research Foundaton, PolG Foundation, and Children’s Cardiomyopathy Foundation have joined the Barth Syndrome Foundation in stressing the critical need for this treatment and the lack of alternatives. They collectively urged the FDA to approve elamipretide, emphasizing its potential to significantly enhance the quality of life and survival for patients with Barth syndrome.
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About Barth Syndrome Foundation (BSF)
Barth Syndrome Foundation and our international affiliates comprise the only global network of families, healthcare providers, and researchers solely driven by the mission to save lives through education, advances in treatments and finding a cure for Barth syndrome. Considered a role model in rare disease advocacy, BSF has funded $6.6M USD and catalyzed over $37.7M USD in funding from other sources to advance global scientific discoveries to end the suffering and loss of life from Barth syndrome. Additionally, BSF provides a lifeline to families and individuals living with Barth syndrome around the world, offering individualized support, educational conferences, a patient registry and collaborations with specialist healthcare providers to define standards of care, treatment and rapid diagnosis.
