US-based clinical-stage biotechnology company Stealth BioTherapeutics Inc. announced on Monday that it has completed the mid-cycle review meeting with the US Food and Drug Administration related to its New Drug Application (NDA) for elamipretide intended for the treatment of Barth syndrome, a serious and potentially fatal progressive genetic disorder.

If elamipretide is approved, it will be the first therapy for Barth syndrome. It is also in phase three development for primary mitochondrial myopathy, with pivotal data likely to be revealed in late 2024, and dry age-related macular degeneration.

The FDA has confirmed that it intends to conduct an advisory committee meeting, which Stealth expects to occur this autumn.

The NDA for elamipretide received priority review and has been granted a Prescription Drug User Fee Act (PDUFA) action date of 29 January 2025.