FDA Reverses Course on UniQure Huntington's Therapy, Accepts Existing Trial Data

The U.S. Food and Drug Administration has reversed its earlier position on UniQure's Huntington's disease therapy, agreeing to accept existing trial data for an accelerated review. The decision comes after the FDA previously indicated the data was insufficient and described the treatment as a "failed product."

The regulator has informed UniQure that a three-year analysis from an early-to-mid-stage trial would be sufficient as the main basis for a marketing application under the accelerated approval pathway. Data from the trial published last year showed patients who received a high dose of the therapy, AMT-130, saw a 75% reduction in disease progression.

UniQure plans to submit its application in the third quarter of 2026. The FDA has also agreed to work with the company to finalize the design of a required confirmatory study before the filing, including using patients on standard treatment as a comparison instead of a sham procedure.

Earlier this year, the FDA had demanded UniQure run an entirely new clinical trial with one arm of patients receiving a sham surgery, a fake version of the real procedure. The agency's proposal drew scrutiny because some patients would undergo invasive brain surgery, including drilling small holes into the skull, without receiving the actual therapy, raising ethical concerns among patient groups and experts.

The latest decision potentially reflects the new FDA's appreciation of the challenges in rare disease following the overhaul of its leadership. The agency appears to be trying to repair relations with the rare-diseases sector, with the acting FDA Commissioner meeting with rare disease advocacy groups earlier this month.

Huntington's disease is a fatal inherited neurodegenerative disorder with no approved therapies that slow progression.