BERLIN (dpa-AFX) - Merck KGaA (MKGAY.PK) said the Phase IIIb SPARK study has met its primary endpoint.
The results of the first 26 weeks of this study demonstrated that the addition of Kuvan (sapropterin dihydrochloride) to a phenylalanine-restricted diet in children less than 4 years of age, who have phenylketonuria or PKU and have been previously shown to be responsive to Kuvan, significantly increased tolerance to phenylalanine compared with a phenylalanine-restricted diet alone.
PKU is an inborn metabolic disorder that causes the toxic accumulation of phenylalanine, an essential amino acid found in all protein-containing foods, in the brain and blood.
SPARK was requested by the European Medicines Agency 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.
The 26-week results will be submitted for presentation at upcoming international scientific meetings and for publication in a peer-reviewed journal.
The long-term efficacy and safety of Kuvan will be assessed in the study's 3-year extension period, during which patients will be offered to receive Kuvan in addition to the phenylalanine-restricted diet.
Copyright RTT News/dpa-AFX