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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In an interview with the Pink Sheet, Stealth’s CEO says she is not confident the company could design a clinical trial in the rare disease that would meet the FDA’s expectations.
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