Iptacopan

**Semantic type:** Pharmacologic Substance

**Definition:** An orally available, small-molecule inhibitor of complement factor B (FB) with potential immunomodulatory activity. Upon administration, iptacopan binds to FB and prevents the formation of the alternative pathway (AP) C3-convertase (C3bBb). This limits the cleavage of C3 to the active fragment C3b and may prevent C3b-mediated extravascular hemolysis in certain complement-driven disorders such as paroxysmal nocturnal hemoglobinuria (PNH).

**Synonyms:** - Factor B Inhibitor LNP023 - IPTACOPAN - LNP 023 - LNP-023 - LNP023