BioMarin Pharmaceutical Inc announced that its partner Merck Serono, a division of Merck KGaA, Darmstadt, Germany, has received a positive opinion for Kuvan (sapropterin dihydrochloride) as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMEA). The CHMP recommendation will be considered by the European Commission, which will deliver its final decision on the granting of marketing authorization within 67 days.

"The positive opinion from the CHMP is encouraging and bodes well for potential approval, which is on track by the end of the year, triggering a $30 million milestone payment to BioMarin," said Jean-Jacques Bienaime, chief executive officer of BioMarin. "We are excited to work with our partner Merck Serono to offer the first therapeutic option to manage PKU in the European market."

Kuvan (sapropterin dihydrochloride) tablet is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet.

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase.

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.

Merck Serono is the division for innovative prescription pharmaceuticals of Merck, a global pharmaceutical and chemical group. Headquartered in Geneva, Switzerland, Merck Serono discovers, develops, manufactures and markets innovative small molecules and biopharmaceuticals to help patients with unmet medical needs.