Stealth BioTherapeutics Inc. and the US Food and Drug Administration appear unable to agree on the clinical data needed to review a treatment for Barth syndrome, in what may be another example of tension between a sponsor and the agency over how to apply regulatory flexibility for rare diseases.
Impasse At US FDA Could Mean Stealth Abandons Barth Syndrome Treatment
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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