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Danicopan is a first-in-class, oral small molecule inhibitor of complement factor D, developed as an add-on therapy for adults with paroxysmal nocturnal hemoglobinuria (PNH) experiencing extravascular hemolysis (EVH) despite treatment with C5 inhibitors such as ravulizumab or eculizumab. By reversibly binding to complement factor D, danicopan selectively inhibits the alternative pathway of the complement system, preventing formation of C3 convertase and subsequent C3 fragment opsonization that leads to EVH. This mechanism allows for control of both intravascular and extravascular hemolysis in PNH when used in combination with terminal pathway inhibitors. Danicopan is marketed under the brand name Voydeya[1][2][4][5][6][7][8].
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