Seven years after Stealth BioTherapeutics landed a fast track designation from the FDA, the agency has finally agreed to review the company’s application for an ultra-rare, genetic Barth syndrome treatment.
Known as elamipretide, the drug will face an FDA advisory committee. The application won’t get a priority or accelerated review from the agency, the Massachusetts-based company said Monday.
The adcomm is likely to feature a tough discussion and vote because there are no other approved treatments for the fatal disease, and Stealth is submitting data from an open-label extension compared to a retrospective natural history study, and additional supporting efficacy and safety data from a natural history study.
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