Novartis' First-in-Class Oral PNH Drug Iptacopan (LNP023) Designated for Breakthrough Therapy in China

On February 27, the CDE website showed that Novartis' LNP023 capsule (iptacopan) is proposed to be included in the breakthrough treatment category for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a complement-mediated chronic rare blood disorder. According to statistics, the incidence of PNH is approximately one to two per million, with a higher incidence in Asian populations compared to Europe and America. It can occur at any age but is commonly seen in people aged 30-40. Anti-complement C5 therapy (Eculizumab or Ravulizumab) has been internationally recognized as the standard treatment for PNH. However, after anti-C5 treatment, a significant number of patients still experience residual anemia, fatigue, and transfusion dependence, severely affecting their quality of life.

Iptacopan is a first-in-class, oral inhibitor targeting factor B of the alternative complement pathway, developed by Novartis. The product acts upstream of the terminal C5 pathway, simultaneously controlling both intravascular and extravascular hemolysis, addressing the limitations of anti-C5 antibodies while providing patients with an oral monotherapy option.

In addition to PNH, Iptacopan is currently in critical research phases for many other complement-mediated diseases (CMD), including renal diseases such as C3 glomerulopathy (C3G), IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), membranous nephropathy (MN), lupus nephritis (LN), as well as immune thrombocytopenic purpura (ITP) and cold agglutinin disease (CAD).

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