FDA grants long-awaited approval to Stealth’s Barth therapy

At long last, Barth syndrome patients have an approved therapy in the U.S., and critically, Stealth has received a priority review voucher.

The decision to grant accelerated approval to Stealth BioTherapeutics Inc.’s Forzinity elamipretide came after an unusually quick review for the rare disease therapy. A resubmission this summer followed a years-long effort that culminated in a face-to-face meeting with FDA Commissioner Marty Makary. FDA’s acceptance of the resubmitted NDA typically would have meant a six-month review; the agency said its goal was to issue its decision within a little more than a month...