Following tireless advocacy, Carter celebrates FDA approval for Barth syndrome treatment

WASHINGTON, D.C. - Rep. Earl L. "Buddy" Carter (R-GA) today celebrated the Food and Drug Administration's (FDA) announcement that it has granted an accelerated approval for Stealth Biotherapeutics' Barth syndrome treatment, Forzinity (elamipretide HC1).

This landmark decision comes after tireless advocacy from Rep. Carter, who sent a letter to FDA calling on it to approve the drug, which is saving lives in his home state of Georgia. Barth syndrome is an exceedingly rare disease, impacting 150 known individuals in the United States and at least six individuals in the state of Georgia.

"Lives will be saved because of the FDA's decision today. Under President Trump's leadership, we are providing a lifeline to patients with Barth syndrome and their families, who have waited long enough for the hope and relief that Forzinity provides. Today's approval should encourage other makers of treatments for rare diseases to continue to fight to bring their drugs to market, as there is a successful pathway to do so," said Rep. Carter.


"Our family is deeply grateful for Congressman Carter's leadership and unwavering advocacy for the approval of elamipretide. His genuine care for the rare disease community, along with his ability to bring both sides of the aisle together for the common good, has been extraordinary. We are equally thankful for the Congressman and his incredible staff, whose support has meant so much to us," said Haley Bower, aunt of Hope Filchak who currently takes elamipretide through expanded access program.

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