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8h ago

FDA Demands Placebo-Controlled Trial for UniQure’s Huntington’s Gene Therapy

A senior official publicly rejected UniQure’s external-control evidence to set expectations for randomized data.

FILE – The Food and Drug Administration seal is seen at the Hubert Humphrey Building Auditorium in Washington, April 22, 2025. (AP Photo/Jose Luis Magana, File)
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Overview

  • On a media call, a senior FDA official confirmed the agency asked UniQure to run a randomized, placebo-controlled study to demonstrate clinical benefit.
  • The official said the company’s external-control comparison was insufficient and labeled the therapy a “failed” treatment for Huntington’s disease.
  • The FDA disputed claims of a required 10-hour sham brain surgery, saying a control could involve brief anesthesia with one to three small scalp nicks.
  • Regulators said Huntington’s reviews have historically required randomized, placebo-controlled evidence, while UniQure argues such a trial would be infeasible or unethical.
  • HHS spokesman Andrew Nixon called a UniQure statement false, UniQure shares dropped to about $9 from roughly $15 earlier in the week, and investors are scrutinizing FDA consistency on rare-disease approvals.