Neurocrine Biosciences, Inc. announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for valbenazine as a treatment for Huntington disease (HD). The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation. In December 2021, Neurocrine Biosciences reported top-line data from its Phase 3 KINECT-HD study evaluating the efficacy, safety and tolerability of valbenazine, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor being investigated as a once-daily treatment in adults with chorea associated with HD. "Receiving an FDA Orphan Drug Designation validates our continued commitment to developing new treatment options that could benefit the lives of patients living with rare diseases, including those impacted by HD," said Kevin Gorman, Ph.D., Chief Executive Officer. "We are in the process of completing data analysis from the KINECT-HD and the ongoing KINECT-HD2 studies, which will form the basis of our supplemental new drug application (sNDA) for submission to the FDA later this year." Enrollment is ongoing in the KINECT-HD2 open-label study to evaluate the long-term safety and tolerability of valbenazine for the treatment of chorea in Huntington Disease.
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