FABHALTA

Iptacopan is a proximal complement inhibitor that targets Factor B (FB) to selectively inhibit the alternative pathway. In paroxysmal nocturnal haemoglobinuria (PNH), inhibition of FB in the alternative pathway of the complement cascade prevents the activation of C3 convertase and the subsequent formation of C5 convertase to control both C3-mediated extravascular haemolysis (EVH) and terminal complement-mediated intravascular haemolysis (IVH). In C3G, overactivation of the complement alternative pathway leads to deposition of C3 within the glomeruli, triggering inflammation, glomerular injury, and kidney fibrosis. Iptacopan selectively blocks the alternative pathway overactivation by inhibiting the alternative pathway related C3 convertase activity, leading to decreased cleavage of C3 and reduced C3 deposition in the kidney.