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FDA approves first drug for Huntington’s disease
on 17. Aug 2008 in Huntington, Huntington.

Federal drug regulators Friday approved a medication to treat a major symptom of Huntington’s disease, marking the first time since the disorder was first described in a Long Island family 136 years ago that any kind of treatment has been available in the United States.

In Huntington’s, a rare, devastating condition, brain cells degenerate because of a genetic miscue easily passed from one generation to the next. The disorder results in jerky, involuntary movements known as chorea.

The drug tetrabenazine controls the chorea, which affects about 90 percent of people with the disease. It was approved under the Food and Drug Administration’s orphan products program, which is aimed at developing treatments for conditions affecting fewer than 200,000 people. Huntington’s disease affects 30,000 people nationwide.

“I think this is a big deal both in terms of having something to offer patients with Huntington’s disease and … because people have been trying to find something for Huntington’s patients for decades,” said Dr. Andrew Feigin, a Huntington’s researcher at the Feinstein Institute of Medical Research in Manhasset, part of the North Shore-Long Island Jewish Health System.

Feigin, who studied the drug in the clinical trial, added that tetrabenazine “has been around for many years and in other countries, and we knew that it improved chorea.”

“It was a matter of a company being interested in it″ and ultimately taking steps to get it to market, Feigin said.

Tetrabenazine is produced by Prestwick Pharmaceuticals in Washington, D.C.

Doctors have been aware of tetrabenazine since the late 1950s and have used it to treat other movement disorders. Regulators in Europe and Canada approved tetrabenazine in the 1990s for Huntington’s chorea.

In the U.S., advocates for patients say, major pharmaceutical companies show little interest in developing treatments for orphan diseases because they do not have millions of patients who can drive profits.

“It’s just wonderful to have a drug approved by the FDA,” said Dr. Fred Marshall of the University of Rochester, who led the clinical trials. “It’s also important symbolically for the patients and their families. They’ve suffered for so long. The big victory here is putting the drug on the map.”

He emphasized that tetrabenazine is not perfect, and too high a dose can lead to depression and suicidal thoughts and exacerbate chorea.

Huntington’s disease was first described in 1872 by Dr. George Huntington, of Columbia University, who studied an East Hampton family affected by the disease over several generations.

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