Ask Congress to Contact HHS and FDA Regarding Elamipretide

Recently, the FDA rejected the new drug application for the use of elamipretide for Barth syndrome, an ultra-rare form of mitochondrial disease that causes muscle weakness and heart dysfunction. This rejection came despite a 10–6 vote by an FDA Advisory Committee vote in favor of approval. The FDA is instead proposing a longer, uncertain path that patients may not survive to see.

Ask your members of Congress to contact the U.S. Department of Health and Human Services and the U.S. Food and Drug Administration to express concern over the FDA’s decision not to approve elamipretide and encourage a thorough review of the FDA’s rare disease therapy review process to ensure it is fair, transparent, and responsive to patient needs.