sapropterin

Ligand id: 5276

Name: sapropterin

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 8
Hydrogen bond donors 6
Rotatable bonds 2
Topological polar surface area 119.64
Molecular weight 241.12
XLogP -1.18
No. Lipinski's rules broken 1

Molecular properties generated using the CDK

No information available.
Summary of Clinical Use
This drug is used alongside a low-phenylalanine diet to treat patients with phenylketonuria [1], as it acts to control blood levels of phenylalanine.
Mechanism Of Action and Pharmacodynamic Effects
Phenylalanine hydroxylase (PAH) deficiency causes phenylketonuria (characterised by elevated blood phenylalanine). Supplying additional BH4 (a PAH co-factor) is predicted to optimise residual PAH activity, to bring about a reduction in circulating phenylalanine. Elevated phenylalanine levels are associated with the intellectual and developmental disabilities observed in phenylketonuria patients.

PAH-catalysed conversion of phenylalanine to tyrosine is required for the production of epinephrine (adrenaline) and the synthesis of the neuro-transmitters, serotonin, dopamine, and norepinephrine (noradrenaline). It is also involved in apoptosis and other cellular events mediated by nitric oxide production.
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