Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients < 4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial

Muntau A. C., Burlina A., Eyskens F., Freisinger P., De Laet C., Leuzzi V., ...More

ORPHANET JOURNAL OF RARE DISEASES, vol.12, 2017 (SCI-Expanded) identifier identifier identifier


Background: Sapropterin dihydrochloride, a synthetic formulation of BH4, the cofactor for phenylalanine hydroxylase (PAH, EC, was initially approved in Europe only for patients >= 4 years with BH4-responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan r) trial was to assess the efficacy (improvement in daily phenylalanine tolerance, neuromotor development and growth parameters), safety and pharmacokinetics of sapropterin dihydrochloride in children < 4 years.