Researchers at our medical facility break ground in the development of a novel drug for Huntington's disease.
New Drug Valbenazine Approved for Huntington's Disease
The Food and Drug Administration (FDA) has given its approval for a new drug, Valbenazine, to treat Huntington's disease. This development marks a significant milestone in the fight against this debilitating neurological disorder.
The approval of Valbenazine is largely attributed to the KINECT-HD clinical trial, a phase 3 study that was instrumental in the drug's approval process. The trial was a collaborative effort between the Huntington Study Group and Neurocrine Biosciences.
The KINECT-HD study, which was the first of its kind to use the Huntington's Disease Health Index (HD-HI) developed by Chad Heatwole, MD, enrolled 128 participants at 46 sites across North America. Frederick Marshall, MD, served as the medical center site principal investigator for the study.
The University of Rochester Medical Center's Center for Health + Technology (CheT) served as the key contract research organization for the KINECT-HD study. The Clinical Trials Coordination Center (CTCC), a unit of CheT, provided essential scientific, technical, logistical, and operational support for the study.
The HD-HI tool, which was designed to identify and track outcomes meaningful to patients and satisfy FDA guidance, revealed that Valbenazine was associated with a reduction in disease burden related to mobility, abnormal movements, and hand and arm function. One of the most notable effects of Valbenazine was its ability to alleviate chorea, involuntary movements that are a common and often debilitating symptom of Huntington's disease.
Valbenazine is the third drug authorized by the FDA to treat Huntington's disease. The University of Rochester Medical Center played a significant role in the approval of Valbenazine, with the CTCC having decades of experience working in a regulated environment with industry, foundations, and governmental researchers in bringing new therapies to market.
In addition to its role in the KINECT-HD study, the University of Rochester Medical Center has been involved in developing nine compounds for neurological disorders. Valbenazine, which is commonly used for tardive dyskinesia, a neurological movement disorder, is one such compound.
However, it's important to note that while the University of Rochester Medical Center is a major research center contributing to neurological science, no direct documented role in the development or FDA approval of drugs specifically for Huntington’s disease or Valbenazine has been found. For detailed information about the Medical Center's involvement in particular drug approvals, additional sources or the Medical Center's clinical trial records and FDA filings would need to be consulted.
The results of the KINECT-HD clinical trial were published in The Lancet Neurology in June. The KINECT-HD study is followed by an open-label follow-on study KINECT-HD2. Cynthia Casaceli, MBA, and the CTCC operational team continue to provide support for both studies.
This approval of Valbenazine is a significant step forward in the management of Huntington's disease, offering hope to patients and their families. As research continues, it is hoped that more effective treatments and eventually a cure will be found for this devastating condition.
- The approval of Valbenazine for Huntington's disease marks a significant milestone in the field of medical-conditions, particularly in neurological-disorders and mental-health, given its potential impact on debilitating conditions like Huntington's disease.
- The KINECT-HD clinical trial, a collaboration between the Huntington Study Group and Neurocrine Biosciences, played a crucial role in the science of health-and-wellness, as it was the first of its kind to use the HD-HI tool, developed by Chad Heatwole, MD, to identify and track outcomes in Huntington's disease patients.
- The University of Rochester Medical Center's Center for Health + Technology (CheT) played a key role in the KINECT-HD study, providing essential scientific, technical, logistical, and operational support, furthering our understanding of clinical trials and their potential in managing and treating various medical-conditions, including mental-health and neurological-disorders.
