danicopan + C5 inhibitor

Danicopan (Voydeya) in combination with a C5 inhibitor (eculizumab or ravulizumab) is a therapeutic regimen developed by Alexion Pharmaceuticals (AstraZeneca) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Danicopan is a first-in-class, oral small molecule inhibitor of complement Factor D, which acts in the alternative pathway of the complement system. While C5 inhibitors effectively manage intravascular hemolysis in PNH, approximately 10-20% of patients experience clinically significant extravascular hemolysis (EVH) due to C3 opsonization of red blood cells. By inhibiting Factor D, danicopan prevents the formation of C3 convertase, thereby reducing EVH. This combination therapy was approved by the FDA in May 2024 based on the pivotal ALPHA Phase III trial (NCT04469465), which demonstrated significant improvements in hemoglobin levels, transfusion avoidance, and quality of life in patients with PNH and EVH despite stable C5 inhibitor therapy.

Brand names

VoydeyaSolirisUltomiris

Other names

danicopan + eculizumabdanicopan + ravulizumab