ARTICLE | Editor's Commentary
Approval of Stealth’s Barth therapy could be bittersweet — a Perspective
Unless priority review vouchers are reauthorized, and FDA reforms put in place, it will be the exception, not the rule
August 25, 2025 11:15 PM UTC
Barth syndrome patients and advocates were elated to meet face-to-face with FDA Commissioner Marty Makary last Friday, capping a years-long effort to secure approval of elamipretide from Stealth BioTherapeutics.
“We secured a face to face with the FDA commissioner #DrMakary,” wrote Amy Bono Wilson, the mother of a 24-year-old with Barth syndrome, in an Instagram post. “To say we are stoked is an understatement.” She added: “THE STARS HAVE LINED UP FOR US! FINALLY.”...