FDA Approves Groundbreaking Barth Syndrome Therapy Following Push by Congresswoman Cammack

Washington, DC — After leading a letter with members of the Florida delegation urging swift action, Congresswoman Kat Cammack (FL-03) is honored to share that the Food and Drug Administration (FDA) has approved the first-ever treatment for Barth syndrome, a rare and life-threatening disease that primarily affects boys. 

“For far too long, families living with Barth syndrome have fought to be heard and often felt invisible. I was proud to lead this effort and thankful the FDA listened and is finally providing life-changing treatment for individuals with this devastating disease,” said Congresswoman Cammack. “Hope is finally here—not just for Barth families, but for the millions of Americans with rare diseases still waiting for a treatment. Thank you to my colleagues for standing with me in pressing the FDA to act.”

Background: 
Barth syndrome is a rare genetic disorder that weakens the heart and muscles, mainly affecting boys. It often causes extreme fatigue, limits daily activities, and can lead to early death.

The newly approved treatment, Forzinity, helps by improving how cells make and use energy. In clinical trials, patients developed stronger muscles, which may help them walk farther, gain independence, and improve their quality of life.

The FDA granted approval through its accelerated pathway and recognized Forzinity as a treatment for rare childhood diseases—underscoring the significance of this breakthrough for families who, until now, had no options.

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