The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.

A US Food and Drug Administration (FDA) advisory committee (AdCom) has voted in support of Stealth Biotherapeutics’ ...

The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome, a rare genetic disorder.

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome.

The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome.

Follows Positive FDA Advisory Committee Meeting Vote Concluding that Elamipretide is Effective for the Treatment of Barth Syndrome – NEEDHAM, Mass., Jan. 23, 2025 /PRNewswire/ -- Stealth ...