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FDA Approves Drug for Treatment of Phenylketonuria

Kuvan is first drug of its kind to treat phenylketonuria

FRIDAY, Dec. 14 (HealthDay News) -- The U.S. Food and Drug Administration announced Thursday that it has approved Kuvan (sapropterin dihydrochloride) for the treatment of tetrahydrobiopterin-responsive phenylketonuria (PKU). PKU is a rare genetic disorder in which an enzyme deficiency leads to build-up of phenylalanine in the body to toxic levels, resulting in mental retardation, seizures and other neurologic complications.

The FDA granted Kuvan a fast-track designation in 2006 since it is the first drug of its kind to treat PKU. Four short-term clinical trials including 579 patients with the disease demonstrated the safety and efficacy of Kuvan. In patients with some residual enzyme function, Kuvan works by increasing the enzymatic breakdown of phenylalanine, resulting in lower blood phenylalanine levels.

Kuvan is to be used in conjunction with a phenylalanine-restricted diet, and patients treated with Kuvan should undergo monitoring of blood phenylalanine levels under the supervision of their physician. Kuvan was developed by BioMarin Pharmaceutical Inc. of Novato, Calif., and Merck Serono, a division of Merck KgaA, Darmstadt, Germany.

"This new drug therapy represents hope for patients and families dealing with this difficult disease," comments Janet Woodcock, M.D., acting director of the FDA's Center for Drug Evaluation and Research. "Now, for the first time, there is a medical intervention to help patients and their families slow the devastating neurological effects of this disease."

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