Merck Serono, BioMarin Submit MAA

Merck Serono, a division of Merck KGaA, has submitted an MAA to the EMEA for sapropterin dihydrochloride (Kuvan in the U.S.) as an oral treatment for patients suffering from significant hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.

The compound is being developed in collaboration with BioMarin Pharma. Acceptance of the MAA filing triggered a $15 million milestone payment to BioMarin. Sapropterin has received orphan medicinal product designation in the European Union. In the EU, orphan medicinal product designation is conferred upon investigational products for diseases that affect fewer than five in 10,000 patients.

“The MAA filing for sapropterin represents a notable milestone in our joint effort with Merck Serono to bring the first treatment option to PKU and HPA patients around the world,” said Jean-Jacques Bienaime, BioMarin’s chief executive officer. “We are working with the FDA on the Kuvan NDA, including label discussions, and gearing up for the anticipated U.S. launch of the product in the fourth quarter of 2007.”