Drugs

FDA Extends Review of Stealth Bio NDA

FDA has extended by three months its review of a Stealth BioTherapeutics NDA for elamipretide, a first-in-class mitochondria-targeted therapeutic for treating Barth syndrome. The new user fee review action target date is 4/29.

The review extension is intended to allow FDA to review supplemental information provided in response to recent requests that were received following the agency’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) 10/10/2024 meeting, which voted 10 to 6 that favored product approval.

In an FDA briefing document released before the meeting, reviewers said the company’s claim that elamipretide reduces pathological apoptosis/necrosis was not evaluated in cells from Barth subjects or in TAZ-deficient mice, the most relevant model of the disease. “Furthermore, no convincing data were provided that demonstrate any improvement of cardiac structure and function in TAZ-deficient mice over a treatment duration that did improve these measures in non-BTHS [Barth syndrome] disease models,” they wrote.

Stealth and the agency have previously discussed the challenges of conducting additional clinical trials in Barth syndrome, especially in 2021 when the agency issued a refuse-to-file letter indicating that the application did not contain a single adequate and well-controlled trial that could establish evidence of effectiveness, according to the briefing document. FDA continued to recommend a new Phase 3 trial, but Stealth told FDA that their intent was to resubmit the NDA without conducting a new trial.

Stealth resubmitted the NDA 1/2024 and FDA says it decided to accept the elamipretide submission “so that it could undergo a more detailed review and be brought to an advisory committee for external input.” 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.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

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