Fabhalta
What Fabhalta is used for
Paroxysmal nocturnal haemoglobinuria Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. Complement 3 glomerulopathy Fabhalta is indicated for the treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated (see section 5.1).
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- PRIME: a priority medicine that the EMA supported early because it may meet an unmet need.
Key facts
- Brand
- Fabhalta
- Active substance
- iptacopan
- Disease area
- Hemoglobinuria, Paroxysmal
- Medicine group
- Immunosuppressants
- ATC code
- L04A
- Marketing authorisation holder
- Novartis Europharm Limited
- Authorised in the EU since
- 2024
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Fabhalta used for?
Paroxysmal nocturnal haemoglobinuria Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. Complement 3 glomerulopathy Fabhalta is indicated for the treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated (see section 5.1).
What is the active substance in Fabhalta?
Fabhalta contains iptacopan.
Who makes Fabhalta?
The EU marketing authorisation holder is Novartis Europharm Limited.
Is Fabhalta an orphan medicine?
Yes. Fabhalta is designated an orphan medicine in the EU, for a rare disease (Hemoglobinuria, Paroxysmal).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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