FDA Approves BioMarin's PKU Treatment

The FDA has approved BioMarin Pharmaceutical’s Kuvan tablets for the treatment of phenylketonuria (PKU).

PKU is a disorder caused by a deficiency of the enzyme pheylalanine (Phe) that can result in brain damage and mental retardation.

Barbara Burton, the clinical investigator in the Kuvan (sapropterin dihydrochloride) Phase II and III trials, said the drug helped control blood Phe levels in PKU patients.

Kuvan is a result of a partnership with Merck Serono.