The first US drug for Huntington’s disease, the genetic brain disorder that killed folk singer Woody Guthrie, won the backing of an FDA advisory panel this afternoon. The committee agreed that Xenazine should be approved, despite the risk of depression, difficulty swallowing and other side effects, Bloomberg News writes. About 30,000 Americans are estimated to suffer from the ailment.

“It’s a drug that needs to be out there on the marketplace, and I think we need to design the follow up of it so it’s watched carefully,” panel member James Couch Jr., chairman of the neurology department at the University of Oklahoma Health Sciences Center, said at the panel meeting in Beltsville,
Maryland. The manufacturer, Prestwick Pharmaceuticals, vowed to control distribution and monitor patients if the agency approves the med.

Huntington’s is an inherited disease that causes nerve cells in the brain to deteriorate, leading to mood swings, forgetfulness and uncontrolled spasms of the fingers, feet or face. There is no cure for the disease, and it usually ends in death from complications, such as pneumonia, falls or suicide. Prestwick, which first sought FDA approval two years ago, already sells Xenazine in Canada under the trade name Nitoman.

The FDA had delayed the drug last year after deciding that docs may confuse the side effects seen in studies with symptoms of Huntington’s. Forty-four of the 111 patients who took Xenazine tablets in Prestwick’s studies reported depression, 20 were unable to remain still and 13 had muscle stiffness or difficulty with coordination, according to the FDA analysis. The agency agreed that the drug controlled involuntary movements.

Caregivers who spoke at the meeting said patients with Huntington’s disease are importing the med from Canada or Europe because they can’t get the drug in the US. They urged the advisory panel to recommend approval so people can obtain and use the drug safely, if they choose. The drug “might not be for every person with HD,” Barbara Boyle, national executive director of the Huntington’s Disease Society of America, told the panel. “But we believe family members have the right to learn about this drug and have the right to make the decision.”

The nonprofit society is based in New York and was founded by the widow of folk singer Woody Guthrie after he died of complications from Huntington’s disease in 1967.

Researchers discovered the genetic mutation that causes Huntington’s in 1993 and can now test for it in people who have symptoms or a family history of the disease. Parents with the malady have a 50 percent chance of passing it on to their children, and all people with the gene eventually develop the
disease if they live long enough.

The age of onset for Huntington’s disease varies. Katharine Moser, a 26-year-old who has tested positive for the mutated gene, told the panel that Xenazine approval would give hope to patients who haven’t yet succumbed to the disease’s effects. “The thought that I am going to lose my independence and be dependent on someone has to be the most depressing fact that I can imagine,” said Moser, an occupational therapist in
Manhattan. “If I have one extra year or even one extra day, it’s worth it.”

Source: Bloomberg News (we apologize for the lack of a link, but will provide one as soon as possible).