Posted by Michael Wonder on 25 Aug 2021
Stealth BioTherapeutics submits elamipretide new drug application to FDA for treatment of Barth syndrome
24 August 2021 - Stealth BioTherapeutics today announced the submission of a new drug application to the U.S. FDA for elamipretide, the company's lead product candidate, for the treatment of Barth syndrome, an ultra-rare genetic condition with no FDA- or EMA-approved therapies.
The submission is based on results from the SPIBA-001 Phase 3 retrospective natural history control trial, which compared data from the open-label portion of the TAZPOWER Phase 2/3 clinical trial to matched natural history controls.
