Stealth Bio confident, despite earlier FDA delays for elamipretide

The US Food and Drug Administration (FDA) has identified a potential path forward for elamipretide for the treatment of Barth syndrome, Stealth BioTherapeutics (OTCQB: MITO) revealed today.

The company noted that this follows years of discussions with the agency and a 2024 Cardiovascular and Renal Drugs Advisory Committee meeting (Ad Com) that concluded that elamipretide is effective for the treatment of Barth syndrome.

Stealth submitted data in its January 2024 new drug application (NDA) showing that knee extensor muscle strength, which improved by over 45% in the company’s Phase II clinical trial, was significantly correlated with improvements on the six-minute walk test, an FDA-recognized indicator of clinical benefit. The company proposed in its NDA and the FDA has now agreed to consider knee extensor muscle strength, for which Stealth previously submitted data, as a potential intermediate clinical endpoint to support accelerated approval.