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Another step in what could be the path to the first Barth syndrome therapy

ARTICLE | Regulation

FDA grants priority review to Stealth’s therapy, removing uncertainty over a priority review voucher needed for economic viability in the ultra-rare disease

By Steve Usdin, Washington Editor

May 7, 2024 9:42 PM UTC

FDA has granted priority review status to an NDA for elamipretide to treat Barth syndrome, reversing a decision to assign the drug standard review status.

The agency accepted the NDA on March 29 following a years-long struggle by Stealth BioTherapeutics Inc. and Barth syndrome patient advocates to persuade FDA to review the therapy.