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

• Published in: Orphanet journal of rare diseases 2017-03, Vol.12 (1), p.47, Article 47
• Main Authors: Muntau, Ania C, Burlina, Alberto, Eyskens, François, Freisinger, Peter, De Laet, Corinne, Leuzzi, Vincenzo, Rutsch, Frank, Sivri, H Serap, Vijay, Suresh, Bal, Milva Orquidea, Gramer, Gwendolyn, Pazdírková, Renata, Cleary, Maureen, Lotz-Havla, Amelie S, Munafo, Alain, Mould, Diane R, Moreau-Stucker, Flavie, Rogoff, Daniela
• Format: Article
• Language: English
• Subjects: Algorithms; Analysis; Biopterins - administration & dosage; Biopterins - analogs & derivatives; Biopterins - metabolism; Biopterins - therapeutic use; Child, Preschool; Children; Clinical trials; Diet; Diet - methods; Female; Health aspects; Humans; Hydroxylase; Infant; Infant, Newborn; Male; Medical research; Motivation; Pharmacokinetics; Phenylalanine; Phenylalanine - administration & dosage; Phenylalanine - blood; Phenylalanine 4-monooxygenase; Phenylalanine Hydroxylase - metabolism; Phenylketonuria; Phenylketonurias - blood; Phenylketonurias - diet therapy; Phenylketonurias - drug therapy; Population; Product development; Rare diseases; Safety and security measures; Sapropterin; Sapropterin dihydrochloride
• ISSN: 1750-1172; 1750-1172
• DOI: 10.1186/s13023-017-0600-x

Sapropterin dihydrochloride, a synthetic formulation of BH , the cofactor for phenylalanine hydroxylase (PAH, EC 1.14.16.1), was initially approved in Europe only for patients ≥4 years with BH -responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) trial was to assess the efficacy (improvement in daily phenylalanine tolerance, neuromotor development and growth parameters), safety and pharmacokinetics of sapropterin dihydrochloride in children