Sapropterin dihydrochloride

• CAT Number: I004598
• CAS Number: 69056-38-8
• Molecular Formula: C9H15N5O3 • 2HCl
• Molecular Weight: 314.2
• Purity: ≥95%
• Synonyms: BH4
• Target: Disease Research Fields
• Solubility: H2O: ≥ 34 mg/mL
• Storage: -20°C
• IUPAC Name: (6R)-2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-3H-pteridin-4-one;dihydrochloride
• InChI: InChI=1S/C9H15N5O3.2ClH/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);2*1H/t3-,4+,6-;;/m0../s1
• InChIKey: RKSUYBCOVNCALL-NTVURLEBSA-N
• SMILES: CC(C(C1CNC2=C(N1)C(=O)NC(=N2)N)O)O.Cl.Cl

Sapropterin dihydrochloride (CAT: I004598) is a synthetic form of tetrahydrobiopterin (BH4), a cofactor involved in the synthesis of neurotransmitters such as dopamine, serotonin, and norepinephrine. It is primarily used as a medication to treat phenylketonuria (PKU), a genetic disorder that impairs the body’s ability to process the amino acid phenylalanine. Sapropterin dihydrochloride helps increase the levels of functional BH4 in the body, thereby improving the metabolism of phenylalanine and reducing its buildup in individuals with PKU. By managing phenylalanine levels, sapropterin dihydrochloride can help prevent or minimize the neurological and cognitive complications associated with PKU.

Overview of Clinical Research

Sapropterin dihydrochloride is a nitric oxide synthase stimulant and a phenylalanine hydroxylase stimulant. Biomarin Pharmaceuticals completed a phase III extension trial for Phenylketonuria (PO) in Germany, Ireland, Italy and United Kingdom (EudraCT2004-004513-41) in 2019.

Reference

[1]. Winn SR et al. High dose sapropterin dihydrochloride therapy improves monoamine neurotransmitter turnover in murine phenylketonuria (PKU). Mol Genet Metab. 2016 Jan;117(1):5-11.
[2]. Somaraju UR et al. Sapropterin dihydrochloride for phenylketonuria. Cochrane Database Syst Rev. 2015 Mar 27
[3]. Jurecki ER et al. Sapropterin Dihydrochloride Mixed With Common Foods and Beverages. Top Clin Nutr. 2014 Oct;29(4):325-331.
[4]. Grange DK et al. Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry. Mol Genet Metab. 2014 May;112(1):9-16