FDA Advisory Committee Supports Stealth BioTherapeutics’ Elamipretide for Barth Syndrome Despite Data Concerns

FDA Advisory Committee Vote:
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in favor of Stealth BioTherapeutics' elamipretide for the treatment of Barth syndrome, an ultra-rare genetic disorder.

Data Concerns:
Despite the positive vote, many committee members expressed concerns about the quality of the data supporting elamipretide, citing variability and lack of well-controlled trials.

Urgent Unmet Need:
The committee recognized the devastating impact of Barth syndrome and the significant unmet medical need, which influenced their decision to support elamipretide despite data limitations.

Previous FDA Rejection:
The FDA had previously rejected Stealth's new drug application (NDA) for elamipretide in 2021, citing the lack of an adequate and well-controlled trial.

Target Action Date:
The FDA is currently reviewing Stealth's application with a target action date of January 29, 2025.