Stealth BioTherapeutics Inc. is gambling that Barth syndrome patients and advocates can help carry its NDA for elamipretide forward despite the US FDA’s recommendation that a second clinical trial be conducted before submission.
Stealth Elamipretide NDA Tests Frontiers Of US FDA’s Flexibility In Rare Diseases
FDA wants another trial in ultra-orphan Barth syndrome, but could not identify a feasible design – so Stealth submitted the NDA anyways, based on a retrospective natural history control trial
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