NBIX - Neurocrine Biosciences valbenazine gets FDA orphan drug status for Huntington disease

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Neurocrine Biosciences' (NASDAQ:NBIX) valbenazine to treat Huntington disease (HD). The company said treatment of chorea associated with HD is within the scope of the orphan drug designation. HD is a rare, genetic disease which causes progressive degeneration of nerve cells in the brain. In December 2021, the company reported that valbenazine met the main goal of a phase 3 trial, dubbed KINECT-HD, to treat chorea associated with HD. "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," said CEO Kevin Gorman. The company noted that enrollment is ongoing in the KINECT-HD2 open-label study to evaluate the long-term safety and tolerability of valbenazine to treat chorea in HD.

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Neurocrine Biosciences valbenazine gets FDA orphan drug status for Huntington disease