FDA official discusses UniQure gene therapy for Huntington's disease
CNBC • • general
Key Points:
- The FDA has requested UniQure to conduct a placebo-controlled trial to prove the efficacy of its gene therapy for Huntington's disease, disputing UniQure's claim that such a study is unethical due to the invasive brain surgery involved.
- An anonymous FDA official criticized UniQure for relying on an external control database instead of conducting a randomized, double-blind placebo-controlled study, suggesting the company is avoiding the correct clinical trial after a previous failure.
- UniQure accused the FDA of reversing its earlier stance on the acceptability of their clinical trial data and condemned the FDA's anonymous comments as irregular and misleading.
- The public disagreement highlights tensions between UniQure and the FDA amid broader scrutiny of the agency's recent drug approval
