A US Food and Drug Administration advisory committee review of Stealth BioTherapeutics Inc.’s elamipretide repeatedly circled back to the question of whether further preapproval data collection would be feasible as well as informative.
Stealth’s Elamipretide: Is A New Preapproval Trial Feasible?
Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.
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