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First Ever Drug for PKU

Med Headlines - Phenylketonuria (PKU) is a genetic disorder that can lead to impaired neurological function causing mental retardation and seizures. Although it is one of a very few genetic disorders controlled by diet, dietary restrictions are dramatic and must be started soon after birth.  Unfortunately, until now, there have been no drugs given approval by the US Food and Drug Administration (FDA) for treatment of PKU.

Yesterday, the FDA announced approval for sapropterin dihydrochloride, marketed under the name Kuvan, to treat PKU in conjunction with a diet that restricts the intake of the amino acid phenylalanine.

A diet low in phenylalanine means no breast milk; no meat, fish, or dairy; and a severely restricted intake of starches. All foods sweetened with aspartame, including diet beverages, must be avoided also. Doctors recommend dietary restrictions for the entire life of the PKU patient.

As part of the FDA approval, BioMarin Pharmaceutical and Merck Serono, partners in the development of the drug, must establish a registry of PKU patients and continue studies of PKU patients taking the drug for as long as the next seven years.

Kuvan helps control phenylalanine levels, thereby protecting intellectual function in babies and young children.

The drug will begin shipping immediately.

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