FDA reverses earlier stance, grants uniQure accelerated approval pathway for Huntington's gene therapy

On June 17, 2026, the U.S. Food and Drug Administration issued a second reversal, allowing biotech company uniQure to pursue an accelerated approval pathway for its gene therapy targeting Huntington's disease.

The move signals a shift toward greater regulatory flexibility, a change noted after the departures of prominent critics Marty Makary and Vinay Prasad from the agency’s advisory landscape.

By granting the accelerated route, the FDA could enable uniQure to bring its therapy to patients more quickly, pending further clinical data to confirm safety and efficacy.