Huntington Disease

Overall, the treatment resulted in dose-dependent trends of improvement on key clinical measures including Total Functional Capacity and Composite Unified Hungtinton's Disease Rating Scale.

AMT-130, uniQure’s investigational gene therapy, is the first therapeutic candidate to receive regenerative medicine advanced therapy designation for Huntington disease.

The original approval for chorea associated with Huntington disease was based on the phase 3 KINECT-HD study, an 128-patient cohort trial in which valbenazine met its primary end point.

Teva’s VMAT2 inhibitor was previously approved in a twice-daily formulation. The once-daily extended-release treatment, marketed as Austedo XR, is expected to be available later in 2023 in doses of 6 mg, 12 mg, and 24 mg.