This report was first published by Endpoints News. To see the original version, click here
Will Huntington’s disease patients be willing to risk a 12-hour sham brain surgery if it means a gene therapy for their fatal condition might ultimately be approved?
That’s the implied question at the heart of uniQure’s dispute with the FDA, as an increasing number of rare disease companies have come under heightened — and often contradictory — regulatory scrutiny. Under FDA Commissioner Marty Makary, the agency has trumpeted efforts to accelerate rare disease drug approvals, while at the same time rejecting such treatments and insisting that they be tested in clinical trials that may not be feasible.
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