Posted by Michael Wonder on 20 Oct 2021
Stealth BioTherapeutics receives refusal to file letter from the FDA
20 October 2021 - Stealth BioTherapeutics today announced that it received a Refusal to File letter from the United States FDA regarding Stealth's new drug application for elamipretide, a mitochondria-targeted therapy for the treatment of Barth syndrome.
The FDA determined, upon its preliminary review, that the new drug application was not sufficiently complete to permit a substantive review.
In the letter, the FDA stated that the new drug application does not contain an adequate and well controlled trial that provides evidence of effectiveness, noting that the SPIBA-201 Phase 2 clinical trial of elamipretide for the treatment of Barth syndrome was negative during the randomised, double-blind portion of the trial and that the FDA does not consider the open label extension of the SPIBA-201 trial to be adequate and well controlled.
