> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Stealth Bio NDA for Barth Syndrome Accepted
- URL: https://www.fdaweb.com/stealth-bio-nda-for-barth-syndrome-accepted/
- Published: 2024-04-08T12:00:00.000Z
- Updated: 2026-09-14T14:27:39.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156731

FDA has accepted for review a Stealth BioTherapeutics NDA for elamipretide for treating Barth syndrome. The submission is based on data from the SPIBA-001 Phase 3 Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial, according to the company. FDA previously granted elamipretide a fast track designation in 2017, orphan drug designation in 2018 and rare pediatric disease designation in 2020\. The agency assigned a standard review for the submission, but the company has asked it to reconsider a priority review. FDA also has indicated that it is currently planning to hold an advisory committee meeting to discuss the application, according to the company.

Barth syndrome is described as an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with a reduced life expectancy, with 85% of early deaths occurring by age five and afflicting mostly males.