FDA sets May 1 decision date for uniQure's Huntington's disease gene therapy after year-long regulatory saga

The U.S. Food and Drug Administration has accepted uniQure's biologics license application for its Huntington's disease gene therapy and placed it on the priority review track, with a May 1 target for a final decision.

If the therapy receives approval, it would become the first treatment shown to modify the course of Huntington's disease, a rare inherited neurodegenerative condition that currently has only symptomatic options.

The decision follows a turbulent year in which the agency initially required a sham‑controlled Phase 3 trial after a Phase 1/2 study reported a 75% slowing of disease progression over three years. A later guidance shift allowed uniQure to file for approval, and an advisory committee meeting may be scheduled before the May deadline.