Pioneering researchers at our medical facility are charting a path for a groundbreaking Huntington's disease drug.
The Food and Drug Administration (FDA) has approved a new drug, Valbenazine, for the treatment of Huntington's disease. This marks the third drug authorized by the FDA for this debilitating neurological disorder.
Valbenazine, marketed under the brand name Ingrezza, was previously approved to treat tardive dyskinesia, a movement disorder often caused by antipsychotic medications. However, its potential in treating Huntington's disease has been a subject of interest in recent years.
The University of Rochester Medical Center (URMC) played a significant role in the approval process of Valbenazine for Huntington's disease. While the exact nature of URMC's involvement in the development of the drug is not fully disclosed in publicly available information, it is known that the Center for Health + Technology (CHeT) at URMC served as the key contract research organization for the KINECT-HD study.
The KINECT-HD clinical trial, a phase 3 study, was conducted in collaboration with the Huntington Study Group (HSG) and Neurocrine Biosciences. The trial enrolled 128 participants at 46 sites across North America. Cynthia Casaceli, MBA, and the Clinical Trials Coordination Center (CTCC) operational team members provided scientific, technical, logistical, and operational support for the study. Melissa Kostrzebski, MS, MBA, currently directs CTCC, a unit of CHeT, which has decades of experience working in a regulated environment with industry, foundations, and governmental researchers in bringing new therapies to market.
The results of the KINECT-HD clinical trial were published in The Lancet Neurology in June. According to the HD-HI tool, developed with the support of HSG, Valbenazine was associated with a reduction in disease burden related to mobility, abnormal movements, and hand and arm function. The drug was also found to alleviate chorea, involuntary movements that are a common and often debilitating symptom of Huntington's disease.
The KINECT-HD study and KINECT-HD2 are ongoing open-label follow-on studies. Researchers with CTCC have played a central role in bringing nine new drugs to market, including pramipexole, entacapone, rasagiline, rotigitine for Parkinson's, tetrabenzine and deutrabenazine for Huntington's, dichlorphenamide for periodic paralysis, and omaveloxolone for Friedreich's ataxia.
While Valbenazine is the ninth compound for neurological disorders that URMC has been involved in developing, its approval for Huntington's disease marks a significant milestone in the fight against this devastating disease. Further research and clinical trials are ongoing to explore the full potential of Valbenazine and other treatments in improving the lives of those affected by Huntington's disease.
- The approval of Valbenazine for Huntington's disease, a neurological disorder, was facilitated by the University of Rochester Medical Center (URMC), with significant contributions from the Center for Health + Technology (CHeT) and the Clinical Trials Coordination Center (CTCC).
- The KINECT-HD clinical trial, a phase 3 study, played a crucial role in Valbenazine's approval for Huntington's disease, as it demonstrated the drug's potential in reducing disease burden, alleviating chorea, and improving mobility, abnormal movements, and hand and arm function.
- URMC's involvement in the development of Valbenazine and other therapies and treatments, including those for neurological disorders, underscores its commitment to advancing health-and-wellness through science and medical-conditions research.
