FDA doubles down: agency demands sham‑controlled trial for uniQure’s Huntington’s gene therapy

The FDA reaffirmed its reviewers’ decision requiring a randomized, sham‑controlled study for uniQure’s AMT‑130 Huntington’s gene therapy, rejecting the company’s objections that such a trial would be unethical or unduly risky. A senior FDA official briefed reporters to clarify the agency’s position — emphasizing the need for internal randomized data when a therapy is invasive and effect sizes are uncertain. UniQure had argued prior agency communications supported external controls; the FDA said its long‑standing policy in Huntington’s disease favors randomized data, prompting a public disagreement that could shape future surgical gene‑therapy approvals.