Danicopan is an inhibitor of Factor D, a protease in the alternative complement pathway (APC; IC50 = 15 nM in a cell-free assay).1 It is selective for Factor D over a panel of 12 serine proteases when used at a concentration of 10 ?M. Danicopan inhibits hemolysis of erythrocytes isolated from patients with paroxysmal nocturnal hemoglobinuria (IC50s = 4-27 nM). Danicopan (200 mg/kg, p.o.) suppresses APC activity in monkeys.1
1.Yuan, X., Gavriilaki, E., Thanassi, J.A., et al.Small-molecule factor D inhibitors selectively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndromeHaematologica102(3)466-475(2017)