FDA Approves First Drug for Huntington's Disease
The U.S. food and Drug Administration approved tetrabenazine, the first drug approved for use in the United States to treat Huntington’s disease. 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.
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.
“This is a huge deal,” said Marshall. “Ninety percent of patients with Huntington’s disease have chorea, and many suffer terribly. It’s hard to describe the torment that these people undergo. We had several patients who experienced a dramatic improvement in their quality of life. Some patients who hadn’t been able to attend church for years were able to do so. Others were able to go out to a restaurant and have a meal for the first time in years. It’s impossible to over-emphasize what this has meant to some patients, who are able to reclaim part of the life that they have lost due to this disease.”
Marshall is part of a team of doctors, scientists and nurses at the University of Rochester Medical Center that treats people from more than 200 families from throughout the Northeast with the disease. He has treated patients with the disease since 1991.
The drug does have some side effects, including the ability to worsen depression and to make movement more difficult. The drug does not appear to help other symptoms of Huntington’s disease, and it does not slow the progression of the disease or stop the underlying disease process. But most neurologists feel that overall, the benefits of the drug outweigh the side effects, especially considering that there has been no medication
Prestwick Pharmaceuticals of Washington, D.C. owns the rights to develop and sell tetrabenazine in the United States. The medication was originally developed in the 1950s to treat psychosis, but was quickly pushed aside by more effective medications. But doctors in the United Kingdom found it to be effective to treat the excessive involuntary movements of Huntington’s.
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