NCIt 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).;
https://www.has-sante.fr/jcms/p_3517015/fr/fabhalta-iptacopan-hemoglobinurie-paroxystique-nocturne 2024 false false false France iptacopan treatment outcome insurance, health, reimbursement paroxysmal nocturnal hemoglobinuria hemoglobinuria, paroxysmal adult guidelines for drug use administration, oral complement inactivating agents orphan drug production anemia, hemolytic Iptacopan Hydrochloride evaluation of the transparency committee Iptacopan
--- https://ansm.sante.fr/tableau-marr/iptacopan 2024 false false false France French guidelines for drug use risk management patients guideline Iptacopan iptacopan
--- https://ansm.sante.fr/tableau-acces-derogatoire/fabhalta 2024 false false false France French Iptacopan iptacopan summary of product characteristics package leaflet guidelines for drug use paroxysmal nocturnal hemoglobinuria adult anemia, hemolytic complement factor B C3 Glomerulonephritis