Iptacopan (LNP023) Mechanism of Action

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disease of hematopoietic stem cells that can lead to hemolysis, thrombosis, and bone marrow failure. A mutation in the PIG-A gene leads to dysregulation of the complement system and immune function. This ultimately causes intravascular and extravascular hemolysis as well as inflammation.1-3

Iptacopan is an oral investigational inhibitor of factor B, a key component of the alternative pathway of the complement system. Preclinical experiments have shown that blockade of this activation pathway with iptacopan may prevent hemolysis and inflammation caused by PNH.3-5

Mechanism of Action Video

Mechanism of Action Video

For information about PNH research from Novartis, please visit the Pipeline Navigator.


Acronyms: MOA = mechanism of action; PIG-A = phosphatidylinositol glycan-class A; PNH = paroxysmal nocturnal hemoglobinuria.

References: 1. Gulbis B, Eleftheriou A, Angastiniotis M, et al. In: Posada de la Paz M, Groft S, eds. Rare Diseases in Epidemiology, Advances in Experimental Medicine and Biology. Springer; 2010;686:389. 2. Takeda J, Miyata T, Kawagoe K, et al. Cell. 1993;73:703-711. 3. Risitano AM. In: Lambris JD, Holers V, Ricklin D, eds. Complement Therapeutics: Advances in Experimental Medicine and Biology. Springer; 2013:155-172. 4. Data on file; LNP023 Investigator's Brochure, Edition 7, 24 March 2020. Novartis. 5. Shubart A, Anderson K, Mainolfi N, et al. Proc Natl Acad Sci USA. 2019;116(16):7926-7931.


Compound(s) are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established and there is no guarantee that they will become commercially available for the use(s) under investigation.

MOA data are based on in vitro/in vivo data.