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Paroxysmal Nocturnal Hemoglobinuria
Iptacopan
Complement Factor B Inhibitor
Table of contents
**Updates**
We updated this drug summary on 21st of April 2025, removed 'Snippets at a glance', removed TLDR section, added simple definitions, and condensed longer paragraphs into summary points to improve readability.
What is iptacopan used for?
Targets Factor B in the alternative complement pathway, reducing red blood cell destruction and kidney damage.
Treats 3 conditions:
PNH - Rare blood disorder where red blood cells (RBC) break down prematurely, causing anaemia and clotting risks.
IgAN - Kidney disease with protein leakage due to immune attacks on kidney filters.
C3G Ultra - rare kidney disease causing inflammation and progression to kidney failure.
Factor B - A protein that activates the immune system’s attack on red blood cells and kidneys.
Alternative complement pathway - Part of the immune system that, when overactive, damages healthy cells.
Anaemia - Low red blood cell count, causing fatigue, pale skin, or shortness of breath.
PNH - Paroxysmal Nocturnal Hemoglobinuria.
IgAN - Primary Immunoglobulin A Nephropathy.
C3G - Complement 3 Glomerulopathy.
What is the mechanism of action of iptacopan?
Blocks the alternative complement pathway, preventing C3 protein buildup in kidneys and RBC destruction.
C3 protein - A complement protein that triggers inflammation when overactive.
What are the contraindications of iptacopan?
Encapsulated bacteria - Bacteria with protective coatings linked to meningitis and sepsis.
Sepsis - Life-threatening systemic infection response.
Meningitis - Inflammation of the brain or spinal cord membranes, often caused by infections.he body’s defence against germs and diseases.
Important iptacopan prescribing safety information.
Cholesterol - A fatty substance in blood; high levels increase heart disease risk.
Triglycerides - A type of fat in blood; elevated levels may harm blood vessels.
REMS - Risk Evaluation and Mitigation Strategy, a safety program for high-risk drugs.
Rebound haemolysis - Sudden return of red blood cell destruction after stopping treatment.
Iptacopan patient counselling advice.
Jaundice - Yellowing of the skin or eyes due to red blood cell breakdown or liver problems.
References
References
- Barratt, J., Rovin, B., Zhang, H., Kashihara, N., Maes, B., Rizk, D., Trimarchi, H., Sprangers, B., Meier, M., Kollins, D., Wang, W., Magirr, A. and Perkovic, V. (2022). POS-546 EFFICACY AND SAFETY OF IPTACOPAN IN IgA NEPHROPATHY: RESULTS OF A RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED PHASE 2 STUDY AT 6 MONTHS. Kidney International Reports, 7(2), p.S236. doi:https://doi.org/10.1016/j.ekir.2022.01.577. Link
- Bomback, A.S., Kavanagh, D., Vivarelli, M., Meier, M., Wang, Y., Webb, N.J.A., Trapani, A.J. and Smith, R.J.H. (2022). Alternative Complement Pathway Inhibition with Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial. Kidney International Reports. doi:https://doi.org/10.1016/j.ekir.2022.07.004. Link
- Carlos and Patel, B.J. (2024). Iptacopan for the Treatment of Paroxysmal Nocturnal Hemoglobinuria. Expert Opinion on Pharmacotherapy, pp.1–9. doi:https://doi.org/10.1080/14656566.2024.2404110. Link
- FDA (2025). Fabhalta Approved for Complement 3 Glomerulopathy. [online] U.S. Food and Drug Administration. Available at: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-adults-complement-3-glomerulopathy-rare-kidney-disease-reduce [Accessed 21 Apr. 2025]. Link
- Novartis (n.d.). HIGHLIGHTS OF PRESCRIBING INFORMATION. [online] Available at: https://www.novartis.com/us-en/sites/novartis_us/files/fabhalta.pdf [Accessed 21 Apr. 2025]. Link