BioMarin Pharmaceutical Inc. announced today that its partner Merck Serono, a division of Merck KGaA, Darmstadt, Germany, has received a positive opinion for Kuvan® (sapropterin dihydrochloride).
Kuvan® (sapropterin dihydrochloride) Tablets is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU) 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 has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan has received seven years of market exclusivity in the United States. In November 2007, Merck Serono submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride as an oral treatment for patients suffering from hyperphenylalaninemia (HPA) due to PKU or BH4 deficiency. If approved in the EU, it will receive 10 years of market exclusivity for this indication.