Prestwick's tetrabenazine shows benefits in Huntington's

New data show Prestwick Pharmaceuticals' investigational therapy tetrabenazine, at dosages adjusted to patient response and side effects, titrated up to 100mg per day, significantly improved clinical outcomes and reduced symptoms of chorea associated with Huntington's Disease, a debilitating neurological disorder marked by incessant involuntary movements for which there is no approved therapy in the USA.

Results of a double-blind, placebo-controlled, pivotal Phase III study were published in the February 14 issue of Neurology, the official journal of the American Academy of Neurology. In the study, the investigators concluded that tetrabenazine significantly reduced patients' chorea burden, improved global outcome scores, and was generally safe and well-tolerated.

Tetrabenazine is a highly-selective and reversible dopamine depletor that works by inhibiting vesicular monoamine transporter 2. Prestwick has filed a New Drug Application to market the product under the trade name Xenazine. The compound has received an Orphan Drug Designation, because it is targeted to a rare disease that affects fewer than 200,000 people, and also has been granted priority review under the Food and Drug Administration's fast track drug development program.