Recently, the Category I innovative drug Iptacopan Hydrochloride Capsules (trade name: FABHALTA) of Novartis Pharma Schweiz AG was approved for marketing by the National Medical Products Administration with priority review and approval, which is applicable for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy.

Iptacopan hydrochloride binds to factor B (FB) in the complement bypass pathway, regulating C3 cleavage, downstream effector production, and amplification of the terminal pathway, controlling C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis. The marketing of this drug provides a new treatment for adult patients with paroxysmal nocturnal hemoglobinuria.