A Phase 2 Open-Label Study Of Danicopan (Ach-0144471) In Patients With Paroxysmal Nocturnal Hemoglobinuria (Pnh) Who Have An Inadequate Response To Eculizumab Monotherapy

Background: PNH is a rare, acquired blood disorder. A somatic mutation in the PIGA gene of one or more hematopoietic stem cells generates a clone of abnormal erythrocytes (RBCs) that lack alternative pathway (AP) regulatory complement proteins CD55 and CD59. This leads to uncontrolled complement activation on affected RBCs and intravascular hemolysis (IVH).