As part of the NDA submission, BioMarin has requested priority review status. Merck Serono expects to file a marketing authorization application for Kuvan with the European Medicines Agency in the third quarter of 2007.
Phenylketonuria (PKU) is a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase, required for the metabolism of phenylalanine, an essential amino acid. The disorder can result in a variety of complications including severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems.
“This NDA filing represents a significant milestone in our PKU program and our efforts to bring the first treatment option to PKU patients,” said Jean-Jacques Bienaime, CEO of BioMarin. “The NDA filing contains data evaluating Kuvan in approximately 650 human subjects in six clinical studies and represents BioMarin’s largest and most comprehensive filing to date. The company is now gearing up for the US launch of Kuvan in late 2007 or early 2008.”