Study of Kuvan meets primary endpoint in PKU patients - Merck KGaA

Merck KGaA has announced that the Phase IIIb SPARK study of Kuvan (sapropterin dihydrochloride) added to a phenylalanine-restricted diet in children less than 4 years of age who have PhenylKetonuria (PKU) and have been previously shown to be responsive to Kuvan, has met its primary endpoint. The results of the first 26 weeks of this study demonstrated that Kuvan significantly increased tolerance to phenylalanine compared with a phenylalanine-restricted diet alone. The safety profile of Kuvan in this population was consistent with the safety profile for Kuvan described in the European Summary of Product Characteristics.

The 26-week results will be submitted for presentation at upcoming international scientific meetings and for publication in a peer-reviewed journal. SPARK was requested by the European Medicines Agency (EMA) as a post-authorization measure. The positive outcome of the study will enable the submission of a regulatory application for a label extension later this year.