Fabhalta
Fabhalta (iptacopan) is a prescription medication used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder characterized by the destruction of red blood cells. As a complement inhibitor, it works by targeting a specific protein in the immune system to help reduce red blood cell breakdown and improve anemia symptoms.
Fabhalta is an oral complement inhibitor used to treat paroxysmal nocturnal hemoglobinuria (PNH) by reducing red blood cell destruction.
Key facts at a glance
- RxNorm
- 2671075
- Generic
- Fabhalta
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- 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.
Fabhalta is FDA-approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adult patients. It may be used as a standalone therapy or in combination with certain other treatments as directed by a healthcare provider.
Fabhalta is a complement inhibitor that works by blocking factor B, a key protein in the alternative complement pathway. By inhibiting this pathway, it helps prevent the destruction of red blood cells (hemolysis) that occurs in PNH.
Fabhalta is an oral medication typically taken twice daily with food. Patients should be monitored for infections and for signs of breakthrough hemolysis. Treatment should be overseen by a hematologist or specialist experienced in treating PNH. Common side effects may include headache, diarrhea, and respiratory infections.
Official drug label
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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