The U.S. Food and Drug Administration today approved tetrabenazine, the first drug approved for use in the United States to treat Huntington's disease, a fatal, inherited neurodegenerative disorder for which there is no cure. The action comes about eight months after an advisory panel unanimously voted to advise FDA to make the medication available to treat the disease.
The drug is already widely used in Europe, Canada and Australia to treat one of the most disabling symptoms of Huntington's disease, involuntary writhing movements known as chorea. Chorea is one of the trademark symptoms of the disease, which directly affects about 30,000 people in the United States today; another 70,000 people who are alive today will be diagnosed with the disease. FDA has classified tetrabenazine as an "orphan drug," since it's targeted to a disease that directly affects fewer than 200,000 people in the nation.
The pivotal study leading to the drug's approval was done by the Huntington Study Group, based at the University of Rochester Medical Center, and was led by Rochester neurologist Frederick J. Marshall, M.D. That study, published in the journal Neurology in 2006, found that the medication cut down involuntary movements on average by about 25 percent, with many patients experiencing a greater improvement. Overall, patients who received the medication were six times as likely to be considered by their doctors to have improved considerably, compared to participants who received a placebo.
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