During a Thursday morning CNBC interview, FDA Commissioner Marty Makary referenced a rare disease gene therapy trial involving a burr hole in patients' skulls for intraventricular injection, where a randomized control trial showed no benefit despite pressures to approve amid procedure-related morbidity risks. Traders interpreted this as targeting uniQure's AMT-130 for Huntington's disease, driving shares down over 22% intraday amid high volume. The comments arose in defending FDA's gene therapy oversight amid rare disease approval debates, echoing prior 2025 FDA feedback on AMT-130 data sufficiency.
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