Chorea in Huntington Disease
Huntington disease (HD) is a hereditary progressive neurodegenerative disorder in which destruction of neuronal cells in the brain results in motor, cognitive, and psychiatric symptoms. Symptoms generally appear between the ages of 30 to 50 and worsen over a 10 to 25-year period. Many patients with HD experience chorea, a troublesome involuntary movement disorder, in which patients develop sudden, irregular, unpredictable, and non-stereotyped movements. Chorea can affect various body parts, and may interfere with speech, swallowing, posture, and gait.
Roughly 90% of the approximately 41,000 patients in the U.S. diagnosed with HD will develop chorea over the course of the disease.
Valbenazine: An Investigational Therapy for Chorea in Huntington Disease
Valbenazine, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. Valbenazine is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors.
In December 2021, we announced top-line data from our Phase 3 KINECT-HD study evaluating the efficacy, safety, and tolerability of valbenazine in adults with chorea associated with HD. We plan to submit a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) in 2022.
Clinical Trials: Chorea in Huntington Disease
Neurocrine Biosciences is currently conducting KINECT-HD2, a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine for the treatment of chorea in patients with Huntington disease (HD).