Stealth BioTherapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has identified a potential path forward for elamipretide, a novel therapy for Barth syndrome, a progressive and lethal pediatric cardioskeletal disease affecting approximately 150 individuals in the US. Following five years of discussions, a positive advisory committee recommendation, and a complete response letter from the FDA, the Agency proposes an accelerated approval pathway requiring resubmission of the elamipretide NDA. The Company will meet with the FDA next month to discuss the proposed post-marketing study, which has been previously expressed alignment by the Agency.
Source: https://www.prnewswire.com/news-releases/stealth-biotherapeutics-announces-path-forward-despite-disappointing-delay-for-ultra-rare-barth-syndrome-302467718.html