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FDA Accepts New Drug Application for Deutetrabenazine for Treatment of Huntington Disease

VMAT2 inhibitor regulates dopamine levels in the brain

A new drug application (NDA) for deutetrabenazine (Teva Pharmaceutical Industries) has been accepted by the FDA for the treatment of chorea (involuntary writhing movements) associated with Huntington disease (HD), a rare and fatal neurodegenerative disorder caused by the progressive breakdown of nerve cells in the brain.

Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter (VMAT2) that is designed to regulate levels of dopamine, a neurotransmitter, in the brain.

The NDA filing was based on positive results from two phase III studies, FIRST-HD and ARC-HD. In the placebo-controlled, randomized FIRST-HD trial, deutetrabenazine reduced chorea in patients with HD. In addition, positive data from the open-label ARC-HD study demonstrated that patients were able to safely convert from tetrabenazine (Xenazine, Lundbeck), currently the only approved HD treatment, to deutetrabenazine overnight with continued control of chorea. The most commonly reported adverse events in the ARC-HD trial included somnolence, falls, and nasopharyngitis.

The FDA granted deutetrabenazine “orphan drug” status for the treatment of HD in November 2014. Orphan drugs and biologics are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S.

HD is characterized by uncoordinated and uncontrollable movements, cognitive deterioration, and behavioral and/or psychological problems. The classic onset of HD symptoms typically occurs in middle age, but the disease also manifests in children and in the elderly. HD is the most common genetic cause of chorea. Disease progression is characterized by a gradual decline in motor control, cognition, and mental stability and generally results in death within 15 to 25 years after the clinical diagnosis.

HD is a genetic disease, passed from parent to child through a gene mutation. Each child of an HD parent has a 50-50 chance of inheriting the HD gene. If a child does not inherit the HD gene, he or she will not develop the disease and cannot pass it to subsequent generations. A person who inherits the HD gene will sooner or later develop the disease. According to the World Health Organization, HD affects approximately five to seven people per 100,000 in Western countries.

Source: Teva Pharma; August 12, 2015.

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