Pharmaprojects R&D Pipeline News Feed
Merck KGaA's Kuvan becomes first European therapy for hyperphenylalaninaemia
9 Dec 2008
Merck KGaA and its Merck Serono division have received EU approval for their monoamine biosynthesis enhancer Kuvan (sapropterin dihydrochloride) for the treatment of hyperphenylalaninaemia (HPA), making it the first and only drug available in Europe for HPA due to phenylketonuria (PKU) or BH4 deficiency. Until now, the only option for PKU patients had been to restrict themselves to a diet with low levels of the amino acid phenylalanine, non-adherence to which could lead to irreversable mental damage.
The approval of Kuvan was based on data collected from two multinational Phase III trials which demonstrated that Kuvan reduced plasma phenylalanine levels and increased patients' phenylalanine dietary tolerance, reducing the need to limit their diet. Adverse events reported were mild to moderate and transient.
Kuvan had received Orphan Drug designation for HPA and as such will receive 10 years of protection for the treatment of this disease. Launch is expected to commence in the first half of 2009. Kuvan has been launched under the name Biopten for over a decade in Japan, where it was developed by Suntory (now Asubio Pharma, part of Daiichi Sankyo).
