The Food and Drug Administration has agreed to decide by late next month whether to approve an ultra-rare disease drug developed by Stealth BioTherapeutics, a significant step after the company claimed it may have to close its doors if an agency endorsement is not made in coming weeks.
The move comes after protracted uncertainty surrounding the fate of the medication, which was developed to treat Barth syndrome, a rare illness that afflicts about 150 people in the U.S. The company and the FDA have struggled to agree on ways to generate enough of the right kind of study data to make the drug available to this tiny population of patients.
Last fall, an FDA advisory panel recommended that the agency should approve the drug. But FDA reviewers, who did not favor approval, subsequently sought additional information from the company and rejected the application in May. At the time, the FDA said it would only consider accelerated approval, which is used for medicines that treat serious conditions and that rely on surrogate endpoints.
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