Danicopan 是一种可口服的小分子 D 因子选择性抑制剂，对人 D 因子具有高结合亲和力，Kd 值为 0.54 nM。 它可抑制补体替代途径 (APC) 的活性，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。

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

Danicopan (ACH-4471) inhibits the proteolytic activity of purified factor D and its natural substrate factor B binding to C3b, blocking the production of Bb fragments in a dose-dependent manner, with an IC50 value of 0.015 μM. Danicopan (ACH-4471) effectively inhibits hemolysis with IC50 values from 0.0040 μM to 0.027 μM (IC90 values from 0.015 μM to 0.11 μM).

1903768-17-1

C26H23BrFN7O3

580.41

ACH-4471

DMSO:260 mg/mL (447.96 mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years