CAS: 1644670-37-0

Iptacopan

Iptacopan (LNP023) is an inhibitor with high affinity to factor B, with a KD value of 7.9 nM and an IC50 value of 10 nM. Cost-effective and quality-assured.

Iptacopan is an oral, small-molecule therapeutic, which is a complement factor B inhibitor that targets the alternative pathway of the complement system. This pathway is a component of the immune system's innate response and, when dysregulated, can contribute to the pathogenesis of various complement-mediated diseases. The mode of action of Iptacopan involves the selective inhibition of complement factor B, a crucial protein in the alternative complement pathway. By precisely targeting this factor, Iptacopan effectively disrupts the formation of the C3 and C5 convertases, thereby reducing the downstream effects of complement activation, such as inflammation and cell lysis. Iptacopan is primarily used for the treatment of rare hematological conditions, such as paroxysmal nocturnal hemoglobinuria (PNH) and certain forms of atypical hemolytic uremic syndrome (aHUS). These disorders arise due to uncontrolled complement activation, leading to hemolysis and thrombosis. The inhibitor’s specific action makes it a promising candidate for efficiently addressing the underlying pathophysiology of these conditions. Ongoing research is evaluating its efficacy and safety across various complement-mediated diseases, aiming to define its full therapeutic potential within this domain.