STAT+: UniQure’s gene therapy continues to slow Huntington’s progression after four years — SkimNews
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- UniQure reported Tuesday that its experimental gene therapy slowed Huntington's disease progression by 44% versus matched participants in a natural-history control group, four years after surgical implantation into patients' brains.
- AMT-130's magnitude of benefit has waned compared with a similar analysis conducted one year earlier, raising durability concerns as the FDA reviews UniQure's marketing application.
- The 44% slowing was not statistically significant, according to the company's new analysis.
- AMT-130 is a one-time gene therapy delivered by surgical implantation into the brain, targeting the neurodegenerative disorder.
- UniQure's marketing application for the therapy is currently under review by the Food and Drug Administration.
Why it matters: UniQure is seeking FDA approval for the first gene therapy targeting Huntington's, a fatal neurodegenerative disease with no disease-modifying treatments. The 44% slowing at four years sounds dramatic, but a waning effect and lack of statistical significance give the FDA leverage to demand more data before greenlighting a one-time brain surgery with permanent effects.
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