ARTICLE | Regulation
Another step in what could be the path to the first Barth syndrome therapy
FDA grants priority review to Stealth’s therapy, removing uncertainty over a priority review voucher needed for economic viability in the ultra-rare disease
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. ...