Iptacopan , is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria.

Trade name Fabhalta

Routes ofMadministration By mouth

Drug class Complement factor B inhibitor.

An oral factor B inhibitor that improves hemoglobin levels in patients with PNH.

It is approved by the US Food and Drug Administration (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria.

A first-in-class medication.

Iptacopan binds to Factor B of the alternative complement pathway and regulates the cleavage of C3, generation of downstream effectors, and the amplification of the terminal pathway.

In PNH, intravascular hemolysis (IVH) is mediated by the downstream membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3b opsonization. 

Iptacopan acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated extravascular hemolysis  and terminal complement mediated intravascular hemolysis.

Iptacopan contains a black box warning for the risk of serious and life-threatening infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B.

In a clinical study with twelve participants, iptacopan as a single drug led to the normalization of hemolytic markers in most patients, and no serious adverse events 

It improves hematologic and clinical outcomes in anti-C5 treated patients with persistent anemia, in whom iptacopan shows superiority to anti-C5 therapy, and in patients who have not received complement inhibitors.

Iptacopan is an oral, proximal complement inhibitor the targets factor be in the alternative pathway and abrogates extravascular hemolysis and maintains intravascular Mall assist control in patients with PNH and persistent anemia, despite anti-C-5 treatment and controls intravascular hemolysis control without causing extravascular hemolysis in patients who had not received the complement inhibitor in the three months before treatment.


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