The marketing authorization is supported by data from two international, double-blind, randomized, placebo-controlled Phase III clinical trials in patients with hyperphenylalaninemia (HPA) due to phenylketonuria (PKU).
According to the company, the Phase III data show that treatment with Kuvan reduces blood phenylalanine levels and increases the proportion of patients with blood phenylalanine levels within target range, resulting in a higher dietary phenylalanine tolerance, which may reduce the need to limit phenylalanine intake in patients’ diet.
Kuvan, which had previously received orphan medicinal product designation from the European Medicines Evaluation Agency (EMEA), is the first drug approved in Europe for HPA due to PKU or BH4 deficiency, the company said.
The European Commission has granted marketing authorization for the 27 countries of the EU as well as Iceland, Liechtenstein and Norway. As an orphan medicinal product and the first drug approved for the treatment of HPA, Kuvan will receive 10 years of data protection in the EU for this therapeutic indication, said Merck Serono. Launch of Kuvan in Europe is expected to start in the first half of 2009.
Roberto Gradnik, executive vice president of commercial Europe at Merck Serono, said: With the approval of Kuvan, Merck Serono provides patients access to an efficient treatment to better control their blood phenylalanine levels. This will contribute to improving their quality of life and may ultimately reduce the risk of lasting mental impairment.