In the latest twist over the fate of an ultra-rare disease drug, Stealth BioTherapeutics said it received “conflicting” signals from the U.S. Food and Drug Administration about information needed to approve its therapy and, consequently, is making contingency plans to close the company.
At issue is a treatment for Barth syndrome, a rare illness that causes an enlarged heart, muscle weakness, and a shortened life expectancy. The disease afflicts up to 150 people in the U.S., an extremely small number that has, at times, made it difficult for Stealth and the FDA to agree on ways to generate enough of the right kind of study data to make the drug available to patients.
For the past few years, the company has traveled a complicated path as the agency bounced its marketing application between different divisions. Winning FDA approval is rarely easy, but the sequence of events was sometimes cited by patient advocacy groups as an example of the need to convince the agency that approvals for rare disease drugs require more flexibility.
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