FDA accused of demanding 10-hour placebo brain surgeries to approve Huntington's disease therapy

FDA accused of demanding 10-hour placebo brain surgeries to approve Huntington’s disease therapy

US Food and Drug Administration officials have pushed for more sham-controlled trials, including for COVID-19 vaccines.

A US company promising a world-first treatment for Huntington’s disease says it was told it had to conduct 10-hour placebo brain surgeries for a study.

The US Food and Drug Administration (FDA) has already returned serve, saying it actually asked for 30-minute sham surgeries. But the clash puts demands for placebo-controlled trials under the microscope — a preoccupation of key US health bureaucrats, including Robert F Kennedy Jr.

The company, UniQure, says its one-time gene therapy AMT-130 slows Huntington’s disease progression by 75%.

Last year, it said it would seek FDA approval using phase I-II data, including a three-year follow-up of 24 patients compared with an external control group — propensity-matched patients in a Huntington’s disease observational database.

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