货号:A1216730 同义名: ACH-4471;ACH-CFDIS
Danicopan is selective and orally active small-molecule factor D inhibitor, shows high binding affinity to human Factor D with Kd value of 0.54 nM. It has potential to block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
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描述 | The complement system provides an important defense against bacteria, fungi, and viruses. Complement factor D (CFD) is a specific serine protease that cleaves its unique substrate and is a promising therapeutic target to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Danicopan (ACH-4471) is selective and orally active small-molecule factor D inhibitor, shows high binding affinity to human Factor D with Kd value of 0.54 nM. ACH-4471 potently inhibited hemolysis with IC50 values ranging from 0.0040 μM to 0.027 μM, and blocked the APC-mediated killing by aHUS patient sera at sub-micromolar concentrations, which indicates ACH-4471 has the potential as oral therapeutics for human diseases driven by the alternative pathway of complement, including aHUS[1]. In a second study in PNH patients with poor response to eculizumab (defined as the need of red blood cell transfusion), ACH-4471 was given as add-on treatment on top of standard eculizumab at three different doses (100, 150, or 200 mg thrice a day)[2]. |
计算器 | ||||
存储液制备 | 1mg | 5mg | 10mg | |
1 mM 5 mM 10 mM |
1.72mL 0.34mL 0.17mL |
8.61mL 1.72mL 0.86mL |
17.23mL 3.45mL 1.72mL |
CAS号 | 1903768-17-1 |
分子式 | C26H23BrFN7O3 |
分子量 | 580.408 |
别名 | ACH-4471;ACH-CFDIS;ALXN 2040;ACH-0144471 |
运输 | 蓝冰 |
存储条件 |
液体 -20°C:3-6个月-80°C:12个月 粉末 Inert atmosphere,2-8°C |
溶解度 |
DMSO: 120 mg/mL(206.75 mM),配合低频超声助溶,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 |
动物实验配方 |