BioMarin Pharmaceutical Inc. has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Kuvan (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU) being developed in partnership with Merck Serono.

Merck Serono expects to file a Marketing Authorization Application (MAA) for Kuvan with the European Medicines Agency (EMEA) in the third quarter of 2007.

"This NDA filing represents a significant milestone in our PKU programme 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."

At a pre-NDA meeting in October 2006, BioMarin met with the FDA to discuss key information on Kuvan to be included in the NDA. Consistent with the discussions at that meeting, the fully electronic NDA filing includes a comprehensive set of preclinical, clinical and manufacturing related data on Kuvan. As part of the NDA submission, BioMarin has requested priority review status. The criteria for assigning priority review status to an application are similar to those for fast track status, which Kuvan has already received. If the FDA accepts the NDA and grants the request for priority review, the FDA is expected to take action on the application within six months of its submission. Kuvan also has orphan drug designation, which allows for seven years of market exclusivity within the United States following the approval of the marketing application.

Kuvan is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolise Phe.