产品说明书

Sapropterin 2HCl

Print
Chemical Structure| 69056-38-8 同义名 : (6R)-BH4 dihydrochloride;(6R)-Tetrahydro-L-biopterin dihydrochloride;BH4;(6R)-5,6,7,8-tetrahydro-L-Biopterin (hydrochloride);R-THBP Dihydrochloride;Tetrahydrobiopterin Dihydrochloride;Sapropterin dihydrochloride
CAS号 : 69056-38-8
货号 : A701042
分子式 : C9H17Cl2N5O3
纯度 : 99%+
分子量 : 314.169
MDL号 : MFCD00891665
存储条件：

粉末 Inert atmosphere,Store in freezer, under -20°C

液体 -20°C:3-6个月-80°C:12个月
溶解度 :

DMSO: 190 mg/mL(604.77 mM)，注意：DMSO长时间开封后，会吸水并导致溶解能力下降，请避免使用长期开封的DMSO

H2O: 100 mg/mL(318.3 mM)，配合低频超声助溶
动物实验配方：
生物活性
描述 Sapropterin dihydrochloride is a synthetic version of tetrahydrobiopterin, the naturally occurring pterin cofactor that is required for PAH-mediated phenylalanine hydroxylation[3]. It acts as a pharmacological chaperone, reducing blood Phe concentration and increasing dietary Phe tolerance in BH4-responsive patients with PAH (phenylalanine hydroxylase) deficiency[4]. In patients with PKU (phenylketonuria) who are responders, treatment with BH4 can activate residual PAH (Phenylalanine hydroxylase) enzyme, improve the oxidative metabolism of Phe, and decrease Phe blood levels in some patients. Kuvan (sapropterin dihydrochloride) is available as 100 mg oral tablets and the recommended starting dose is 10 mg/kg/day daily[5]. Sapropterin dihydrochloride (BH4) reduces phenylalanine (Phe) levels and improves white matter integrity in a subset of individuals with phenylketonuria (PKU) known as "responders."[6]. When started after disease onset, oral sapropterin treatment inhibits chronic hypoxia-induced pulmonary hypertension at least in part by recoupling eNOS in the pulmonary vasculature of newborn piglets[7].
临床研究
NCT号 适应症或疾病 临床期 招募状态 预计完成时间 地点
NCT02677870 Phenylketonuria Phase 4 Recruiting August 2018 United States, Ohio ... 展开 >> University Hospitals Cleveland Medical Center Recruiting Cleveland, Ohio, United States, 44106 Contact: Lori P Schillaci, MD    216-844-3936    lori-anne.schillaci@uhhospitals.org 收起 <<
NCT00625820 Kidney Disease ... 展开 >> Albuminuria 收起 << Phase 2 Completed - United States, Michigan ... 展开 >> University of Michigan Ann Arbor, Michigan, United States, 48109 收起 <<
NCT00625820 - Completed - -
实验方案
1mg 5mg 10mg

1 mM

5 mM

10 mM

3.18mL

0.64mL

0.32mL

15.92mL

3.18mL

1.59mL

31.83mL

6.37mL

3.18mL
参考文献

[1]Yildiz Y, Dursun A, et al. Partial hydatidiform mole in a phenylketonuria patient treated with sapropterin dihydrochloride. Gynecol Endocrinol. 2017 Jan;33(1):19-20.

[2]unal o, Gokmen-ozel H, et al. Sapropterin dihydrochloride treatment in Turkish hyperphenylalaninemic patients under age four. Turk J Pediatr. 2015 May-Jun;57(3):213-8.

[3]Harding CO. New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride. Biologics. 2010 Aug 9;4:231-6

[4]Muntau AC, Adams DJ, Bélanger-Quintana A, Bushueva TV, Cerone R, Chien YH, Chiesa A, Coşkun T, de Las Heras J, Feillet F, Katz R, Lagler F, Piazzon F, Rohr F, van Spronsen FJ, Vargas P, Wilcox G, Bhattacharya K. International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria. Mol Genet Metab. 2019 May;127(1):1-11

[5]Clinical Review Report: Sapropterin dihydrochloride (Kuvan) [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2017 Sep

[6]Hawks Z, Shimony J, Rutlin J, Grange DK, Christ SE, White DA. Pretreatment cognitive and neural differences between sapropterin dihydrochloride responders and non-responders with phenylketonuria. Mol Genet Metab Rep. 2017 Feb 23;12:8-13

[7]Dikalova A, Aschner JL, Kaplowitz MR, Summar M, Fike CD. Tetrahydrobiopterin oral therapy recouples eNOS and ameliorates chronic hypoxia-induced pulmonary hypertension in newborn pigs. Am J Physiol Lung Cell Mol Physiol. 2016 Oct 1;311(4):L743-L753