Sapropterin
Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), the natural cofactor for phenylalanine hydroxylase (PAH), used to treat phenylketonuria (PKU) and hyperphenylalaninemia in patients with BH4-responsive PAH deficiency. By supplementing or stabilizing the enzyme cofactor, it enhances residual PAH activity, lowering blood phenylalanine levels and allowing dietary liberalization. It is also investigated in cardiovascular disease due to BH4's role in endothelial nitric oxide synthase coupling.
分子量
241.2500 g/mol
LogP
-1.90
TPSA
132.00 Ų
リピンスキーの五則
適合
治療領域
Pharmacokinetics (PK)
Pharmacodynamics (PD)
2D構造
Cite this structure
Embed this structure
SMILES
C[C@H](O)[C@H](O)[C@H]1CNc2nc(N)[nH]c(=O)c2N1
InChI
InChI=1S/C9H15N5O3/c1-3(15)6(16)4-2-11-7-5(12-4)8(17)14-9(10)13-7/h3-4,6,12,15-16H,2H2,1H3,(H4,10,11,13,14,17)/t3-,4+,6-/m0/s1
Molecular Formula
C9H15N5O3
HBD / HBA
6 / 6
回転可能結合数
2
重原子数
17
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
よくある質問
Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), the natural cofactor for phenylalanine hydroxylase (PAH), used to treat phenylketonuria (PKU) and hyperphenylalaninemia in patients with BH4-responsive PAH deficiency. By supplementing or stabilizing the enzyme cofactor, it enhances residual PAH activity, lowering blood phenylalanine levels and allowing dietary liberalization. It is also investigated in cardiovascular disease due to BH4's role in endothelial nitric oxide synthase coupling.
Yes, Sapropterin is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL1201774. Open-access bioactivity database.
- PubChem — National Center for Biotechnology Information (NCBI). CID 135398654. Chemical information database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.
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