October 10, 2024
FDA Advisory Committee Strongly Recommends Approval of Elamipretide for Treatment of Barth Syndrome
Statement from Barth Syndrome Foundation on FDA Advisory Committee Meeting
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 Foundation, 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.
Additional Resources
Visit the Barth Syndrome Foundation advocacy timeline here
Recording of FDA Advisory Committee:October 10th, 2024 (Full Hearing here)
See Comments Submitted to FDA Public Docket (here)
Highlights of AdComm Hearing
- OPH testimonials here
- Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA), explanation about regulatory flexibility when making a decision in the regulatory review process for a rare condition like Barth syndrome here
- AdComm Vote and post-vote rationale by AdComm Members here
Watch September 13th, 2024 Advocacy Essential Training: How to Submit a Letter to the FDA Docket here
Watch August 1st, 2024 Barth Syndrome Biennial Conference 20204: Lessons from Barth Syndrome Clinical Trials here
Watch May 23rd, 2024 Advocacy Essential Community Briefing: FDA Advocacy Committee here
Learn more about July 18th, 2024 Externally Led Patient Focused Drug Development Meeting here
