Iptacopan
Iptacopan is an oral complement inhibitor that targets Factor B in the alternative complement pathway. It regulates C3 cleavage and downstream effector generation, reducing hemolysis in paroxysmal nocturnal hemoglobinuria (PNH). By modulating the amplification of the terminal pathway, it decreases membrane attack complex formation and associated red blood cell destruction.
Iptacopan is an oral Factor B inhibitor used to treat paroxysmal nocturnal hemoglobinuria by reducing complement-mediated red blood cell destruction.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- L04AJ08
- RxNorm
- 2671061
- Generic
- Iptacopan
- Class
- atc-l04
- Brands
- Fabhalta
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Iptacopan is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). It may be used as monotherapy or in combination with standard care for patients who have responded inadequately to other therapies.
Iptacopan selectively binds to Factor B, a key enzyme in the alternative complement pathway. By inhibiting Factor B, it regulates C3 cleavage and prevents the amplification of complement activation, thereby reducing the formation of the membrane attack complex that causes intravascular hemolysis of red blood cells.
Iptacopan is administered orally, offering an advantage over intravenous complement inhibitors. Patients should be monitored for signs of infection due to increased susceptibility to encapsulated bacteria. Vaccination against Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria meningitidis may be recommended before initiating therapy. Liver function tests should be assessed periodically during treatment.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy