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FDA Advisory Committee Recommends Approval of Tetrabenazine for Huntington Disease
"The advisory committee's support of tetrabenazine represents an important advancement for Huntington disease patients, caregivers, advocates and physicians who treat this devastating disease," said George F. Horner III, president and chief executive officer of Prestwick. "We are committed to continuing to work with the FDA to secure full approval of tetrabenazine."
While the FDA is not required to follow the advice of its advisory committees, they generally do.
HD is a devastating neurodegenerative disease that causes progressive movement disorders, cognitive dysfunction and behavioral changes, that is ultimately a fatal condition. Chorea is characterized by excessive, involuntary and repetitive movements which are the most visible and dangerous manifestations of Huntington disease.
"Patients and physicians do not have any approved treatments to alleviate this disorder or any aspect of Huntington disease," said Frederick Marshall, MD, chief of the geriatric neurology unit at the University of Rochester, who presented data on behalf of Prestwick. "We are hopeful that the FDA will adhere to the recommendations made by the committee to quickly bring tetrabenazine to the patients for whom we care."
Chorea affects over 30,000 Americans, interfering with their ability to perform activities of daily living, including dressing, bathing and caring for themselves. Currently, there are no treatments to stop or reverse the onset or progression of HD and there are no FDA-approved drugs for chorea.
Tetrabenazine is an investigational compound for the treatment of chorea associated with HD. A highly selective and reversible centrally-acting dopamine depleting drug, tetrabenazine works by inhibiting a molecule known as VMAT2 (vesicular monoamine transporter 2).
Adverse events reported with tetrabenazine, most of which were addressed by titrating down the dose, included depression, akathisia, parkinsonism and sedation. Tetrabenazine's effects are largely reversible and manageable.
The FDA has issued an approvable letter for the compound. Tetrabenazine has been designated as an "orphan drug" by the FDA and has been granted "fast track" status.
Source: Prestwick Pharmaceuticals