Recently, the Catogory 1 innovative drug Iptacopan Capsules (trade name: Fabhalta/飞赫达) of Novartis Pharma Schweiz AG was approved for marketing through the priority review and approval procedure by China NMPA. It is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not been previously treated with a complement inhibitor.
Iptacopan through binding with Factor B (FB) in the complement alternative pathway, regulates the cleavage of C3, prevents downstream effectors' generation and terminal amplification loop, so as to control extravascular hemolysis mediated by C3b and intravascular hemolysis mediated by end-complement. The marketing of this drug provides new treatment options for adult patients with PNH.