FDA Advisory Committee Supports Stealth's Elamipretide Despite Data Concerns

The FDA Advisory Committee voted 10-6 in favor of Stealth BioTherapeutics' elamipretide for treating Barth syndrome, an ultra-rare genetic disorder, despite significant variability in the data and the absence of well-controlled trials[1][2]. The committee noted the urgent unmet need for treatments and considered the positive patient testimonies and trends in patient outcomes as persuasive factors in their decision[1]. Despite this support, the FDA has expressed reservations about the drug's approvability, highlighting concerns about the efficacy data, which did not meet the primary endpoint in trials, and noting potential biases and confounding factors[2]. The decision from the FDA is expected by January 29, 2025[1][2].