FDA panel supports approval of Stealth’s elamipretide

Stealth BioTherapeutics (Nasdaq: MITO) has received a favorable vote from a US Food and Drug Administration advisory committee regarding its lead product, elamipretide.

The candidate is being developed for the treatment of Barth syndrome, a rare and life-threatening mitochondrial disease affecting around 150 individuals in the USA.

The cardiovascular and renal drugs advisory committee voted 10 to 6 in favor of the drug's efficacy. Although non-binding, the outcome significantly bolsters the chances of approval for elamipretide, which could become the first therapy approved for Barth syndrome in the USA or Europe.