Danicopan (ACH-4471) 是一种选择性的口服活性 small-molecule factor D 抑制剂，对 small-molecule factor D 具有高结合亲和力，其 Kd 值为 0.54 nM。Danicopan (ACH-4471) 可抑制补体 (APC) 活性的替代途径，具有阻断阵发性夜间血红蛋白尿 (PNH) 和非典型溶血性尿毒症综合征(aHUS) 的补体替代途径的潜力。
货号:ajcx14412
CAS:1903768-17-1
分子式:C26H23BrFN7O3
分子量：580.41
溶解度:DMSO: 260 mg/mL (447.96 mM)
纯度：98%
存储：Store at -20°C
库存：现货

Background:

Danicopan (ACH-4471), a selective and orally active small-molecule factor D inhibitor, shows high binding affinity to human Factor D with Kd value of 0.54 nM. Danicopan (ACH-4471) inhibits alternative pathway of complement (APC) activity, has potential to block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)[1].

Danicopan (ACH-4471) inhibits the proteolytic activity of purified Factor D against its natural substrate Factor B in complex with C3b, blocking production of Bb fragment in a dose-dependent manner with an IC50 value of 0.015 μM[1].Danicopan (ACH-4471)potently inhibits hemolysis with IC50 values ranging from 0.0040 μM to 0.027 μM (IC90 values from 0.015 μM to 0.11 μM) [1].

[1]. Yuan X, et al. Small-molecule factor D inhibitors selectively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Haematologica. 2017 Mar;102(3):466-475.