FDA Defends Demand for New Trial for Rare Brain Disease Therapy

Regulators have asked the company, which says there is already sufficient data, to run a placebo-controlled trial.

The U.S. Food and Drug Administration in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

The Food and Drug Administration is defending its choice not to move forward with reviewing a therapy for a rare brain issue called Huntington’s disease.

A trial of uniQure’s AMT-130 gene therapy is not enough to warrant review of the therapy, a senior FDA official, speaking on condition of anonymity, told reporters on a call on March 5.