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Impasse At US FDA Could Mean Stealth Abandons Barth Syndrome Treatment

 
• By Derrick Gingery

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.

impasse
Stealth Biotherapeutics left a Type A meeting with the FDA not knowing what to do to push the elamipretide development program forward. • Source: Shutterstock

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.

The situation has left Stealth at a crossroads, as it awaits the agency’s response to a request to reconsider the totality of evidence already submitted for elamipretide

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