iptacopan + eculizumab

**iptacopan + eculizumab** is a combination therapy for paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening hematologic disorder. Iptacopan is a first-in-class oral, selective inhibitor of complement factor B, which blocks the alternative complement pathway, thereby controlling both intra- and extravascular hemolysis. Eculizumab is a monoclonal antibody that inhibits complement component 5 (C5), preventing formation of the membrane attack complex and reducing intravascular hemolysis. This combination is used particularly for patients who have insufficient response to eculizumab monotherapy, due to persistent extravascular hemolysis. The combination has been shown in clinical trials to provide greater improvement in hemoglobin levels and reduce transfusion needs in PNH patients with suboptimal response to C5 inhibition alone[1][7].