FDA Reversed Course on uniQure's Huntington's Disease Treatment After Agreeing to Accept Application

The FDA's handling of uniQure's experimental Huntington's disease gene therapy drew attention over the past year after the agency reportedly agreed to accept an application for the treatment, then withdrew that agreement, and later reversed itself again. A former FDA senior official quoted by the Washington Examiner described the initial reversal as "evil." The episode is one of a series of cases that critics say illustrates inconsistent agency behavior toward rare-disease treatments.

The Washington Examiner op-ed, written by healthcare consultant Matt Cover, framed the uniQure case not as an isolated incident but as evidence of a systemic problem. Cover argued that the FDA's tendency to adhere rigidly to standard protocols imposes unnecessary delays on rare-disease innovation without a corresponding safety benefit. He noted that patient advocates for Huntington's disease families eventually had to escalate their case to the White House to prompt the agency to honor its written commitments.

Cover drew on the history of drug repurposing to argue that approvals for rare conditions carry broader downstream value. He cited ibuprofen, gabapentin, and GLP-1 drugs such as Ozempic as examples of treatments initially approved for narrow indications that later became widely used for other conditions. He also noted that exon-skipping therapies for Duchenne Muscular Dystrophy, currently on the market under accelerated approval, face additional regulatory demands that leave their long-term availability uncertain.