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# Panel to Review Stealth Bio Barth Syndrome NDA
- URL: https://www.fdaweb.com/panel-to-review-stealth-bio-barth-syndrome-nda/
- Published: 2024-09-06T12:00:00.000Z
- Updated: 2026-09-14T14:40:37.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5157723

*Federal Register* notice: FDA is announcing a 10/10 Cardiovascular and Renal Drugs Advisory Committee meeting to review a Stealth BioTherapeutics NDA for elamipretide hydrochloride injection, indicated 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.

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.

To view the notice, click [here](https://www.govinfo.gov/content/pkg/FR-2024-09-06/pdf/2024-20062.pdf?ref=fdaweb.com).