US approves Biomarin drug to treat genetic disorder

BioMarin Pharmaceutical Inc won U.S. approval for the first prescription drug to treat phenylketonuria (PKU), a neurological disorder that can cause seizures and mental retardation, federal regulators said on Thursday.

The company expects the drug, called Kuvan, to cost patients $57,000 a year and said it expects it to generate 2008 revenue of $35 million to $70 million.
News of the approval by the U.S. Food and Drug Administration sent BioMarin shares up 5.6 percent in regular trading to their highest level since early 2000.
The stock rallied again after hours, adding another 16 percent after the company announced the drug's pricing.

The drug was developed with Merck Serono, a division of Germany-based Merck KGaA.
The FDA said PKU occurs in one out of every 12,000 to 15,000 live births in the United States.
People with PKU are not able to break down phenylalanine, an amino acid found in protein-rich foods such as meat, dairy and egg products as well as aspartame, a widely used artificial sweetener.

High levels of phenylalanine are toxic to the brain and can lead to mental retardation, behavioral abnormalities, seizures, an inability to focus and organize information, and other neurological complications.

"Now, for the first time, there is a medical intervention to help patients and their families slow the devastating neurological effects of this disease," FDA Deputy Commissioner Janet Woodcock said.

The agency said the safety and efficacy of Kuvan was demonstrated in four short-term clinical studies with 579 patients with the disease.
Kuvan works by increasing phenylalanine hydroxylase enzyme activity, which helps break down phenylalanine and results in lower levels of phenylalanine in the blood, regulators said.
The drug must be used in combination with a diet that restricts phenylalanine.
Shares in BioMarin gained $1.58 to close at $29.76 on Nasdaq, hitting a multiyear high, and leaped to $34.45 in extended trade.

Stock in the Novato, California-based company is up around 80 percent so far this year.

By Lisa Baertlein
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