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sapropterin
- Development stage
- Approved
- Lead developer
- BioMarin Pharmaceutical
- Modality
- Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Allosteric Modulators → Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), a naturally occurring enzyme cofactor. It is used as an adjunct to dietary phenylalanine restriction for the treatment of phenylketonuria (PKU), an inherited metabolic disorder characterized by elevated blood phenylalanine levels due to deficient activity of the enzyme phenylalanine hydroxylase (PAH). Sapropterin works by augmenting and stabilizing residual PAH activity in responsive patients, thereby enhancing the conversion of phenylalanine to tyrosine and reducing toxic accumulation of phenylalanine in the blood. In cases of tetrahydrobiopterin deficiency, it acts as replacement therapy for endogenous BH4. The drug is available in oral formulations and must be used alongside a restricted diet[1][2][3][6][7][8].
- Brand names
- KuvanJavygtor
- Other names
- tetrahydrobiopterinBH4BH-4BH 4sapropterin dihydrochloride
02
Targets
NOS3 (eNOS)TPH1 (Tryptophan 5-hydroxylase 1)TH (Tyrosine hydroxylase)PAH (Phenylalanine hydroxylase)
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