The FDA reversed its stance to allow uniQure to file for accelerated approval of AMT-130, a Huntington's disease gene therapy.
The agency will accept three-year Phase 1/2 study data as the primary submission basis. This shift removes the previous requirement for data from an additional clinical trial.
uniQure shares surged following the June 17 announcement of the regulatory turnaround. The decision establishes a clearer approval pathway for the neurodegenerative disorder treatment.
The company must still finalize a confirmatory study design with the FDA. This development may improve regulatory confidence for the broader gene therapy sector.