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.
BOSTON (October 7, 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, is urging the U.S. Food and Drug Administration (FDA) to approve elamipretide, the first potential therapy for Barth syndrome. The agency’s Cardiovascular and Renal Drugs Advisory Committee is scheduled to review and provide feedback to the FDA related to the New Drug Application (NDA) for elamipretide on October 10.
Barth Syndrome Foundation is a global facilitator for advancing understanding of and developing treatments for Barth syndrome.
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