BCX9930 + ravulizumab

**BCX9930 + ravulizumab** is a combination therapy for paroxysmal nocturnal hemoglobinuria (PNH) involving BCX9930, an oral small molecule inhibitor of complement Factor D, and ravulizumab, a second-generation monoclonal antibody that inhibits complement component C5. BCX9930 blocks the alternative pathway of the complement system, reducing both intravascular and extravascular hemolysis. Ravulizumab, administered intravenously, prevents the cleavage of C5, thereby inhibiting formation of the membrane attack complex that mediates intravascular hemolysis. This combination is investigated for PNH patients inadequately controlled on C5 inhibitors, aiming for broader complement inhibition and improved clinical outcomes[1][2][3][4][5].

Brand names

Ultomiris

Other names

BCX9930 hydrochlorideBCX-9930 hydrochlorideBCX 9930 hydrochloride