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  • 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).
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