STAT+: FDA approves ultra-rare disease drug that became a symbol of uncertainty over regulatory standards

STAT September 19, 2025
Ed Silverman

The agency endorsed a treatment for Barth syndrome, a rare illness that afflicts 150 Americans

After months of uncertainty, the Food and Drug Administration offered accelerated approval for an ultra-rare disease drug that had become a symbol of the fraught balancing act between upholding regulatory standards and accommodating desperate patients and their families.

The agency endorsed a treatment for Barth syndrome, a rare illness that afflicts about 150 people in the U.S. That decision comes after a protracted debate between the manufacturer, a small, privately held company called Stealth BioTherapeutics, and the FDA about how...

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