health

March 5, 2026

FDA official calls UniQure's gene therapy a 'failed' treatment for Huntington's disease

UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.

FDA official calls UniQure's gene therapy a 'failed' treatment for Huntington's disease

TL;DR

  • A senior FDA official stated UniQure's gene therapy for Huntington's disease needs further study to prove its efficacy.
  • The official characterized the treatment as a 'failed therapy' and disputed UniQure's ethical objections to a placebo-controlled trial.
  • UniQure had previously accused the FDA of reversing its stance on the sufficiency of the company's clinical trial data.
  • The company's study used an external control from a database to measure disease decline, which the FDA disputes.
  • UniQure's stock saw a significant drop this year, though it rose on the day of the report.
  • The FDA maintains it never agreed to accept UniQure's 'distorted comparison' and always requires seeing the data.

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