Agency Approves Drug to Treat Genetic Disorder That Can Lead to Retardation

NY Times ^ | December 14, 2007 | ANDREW POLLACK

[neverdem]

The Food and Drug Administration on Thursday approved the first drug to treat a rare genetic disorder that can lead to mental retardation, possibly allowing some people to relax a regimented diet now used to control the disease.

All babies born in the United States are screened for the disease, called phenylketonuria, or PKU. To avoid damage to the brain, people with the disease must adhere to a strict low-protein diet, particularly in childhood but also later in life.

The new drug, called Kuvan, “will be life-changing for some patients,” said Dr. Stephen D. Cederbaum, a professor at the University of California, Los Angeles, who is an adviser to BioMarin Pharmaceutical, the company that developed the drug.

Dr. Cederbaum said that one of his patients, a 25-year-old man who began taking Kuvan as part of a clinical trial, was able to get his first taste of cheese and pizza and to eat enough other foods that “for the first time in his life, he’s not hungry.”

But Dr. Cederbaum said there was also some resistance to the drug because of its price, especially since some patients would get little or no benefit.

BioMarin said that Kuvan would cost an average of $57,000 a year. It would be less than that for small children but could be more than $200,000 for large adults getting the highest dose. The company, based in Novato, Calif., said it expected insurers to pay for the drug.

There are at least 12,000 people in the United States with PKU, according to BioMarin, which collaborated with the German pharmaceutical company Merck on developing the drug...

[skinkinthegrass]

WOW!...the Food and Drug Administration on Thursday approved the first drug to treat a rare common genetic disorder that can lead to mental retardation....

*wish* Ahhh...A cure for Liberalism. */wish*