Prestwick submits application for Huntington’s disease drug

Chorea is the most visible and common of the motor symptoms observed in patients with Huntington’s disease. The condition is characterized by brief, irregular contractions that are not repetitive or rhythmic, but appear to flow from one body part to the next. There is currently no FDA-approved treatment for chorea associated with Huntington’s disease in the US.

Tetrabenazine is a highly selective and reversible dopamine depletor that works by inhibiting vesicular monoamine transporter 2 (VMAT2). Tetrabenazine is approved for marketing outside the US in eight countries and is under regulatory review for approval in several additional countries.

Prestwick’s application is supported by phase III clinical data that suggests tetrabenazine is significantly more effective than placebo in treating patients’ chorea associated with Huntington’s disease, with many patients demonstrating marked to good improvement. The drug has also been shown to be effective in treating children with severe hyperkinetic movement disorders who are resistant to other medications.

The most common side effects with tetrabenazine include sedation, Parkinsonism, depression and insomnia.

The FDA has granted tetrabenazine orphan drug designation to treat a rare disease that affects less than 200,000 patients in the US. The FDA also has designated tetrabenazine a fast track product to facilitate the development and expedite its review as a new drug that’s intended to treat a serious or life-threatening condition, and demonstrates its potential to address an unmet medical need.