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iptacopan

Fabhalta · ATC code L04A

Also known as: İptakopan

Key facts
Active substance
iptacopan
Drug class
Immunosuppressive drug
ATC code
L04A
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
FABHALTA 200 mg hard capsules
Marketing authorisation holder
Novartis Europharm Limited
Information as of
2026-10-28

Key facts

Iptacopan (Fabhalta) is an oral complement factor B inhibitor used to treat paroxysmal nocturnal haemoglobinuria (PNH) and complement 3 glomerulopathy in adults.

What it is used for

  • Monotherapy in adults with PNH who have haemolytic anaemia.
  • Adults with complement 3 glomerulopathy (C3G) in combination with a RAS inhibitor, or when a RAS inhibitor is not tolerated or contraindicated.

Dosing

  • 200 mg orally twice daily, with or without food; in PNH adherence is important to minimise the risk of haemolysis.
  • Missed dose: take one dose as soon as possible, then resume the regular schedule; in PNH monitor for haemolysis after several missed doses.
  • Switching in PNH: start no later than 1 week after the last eculizumab dose or no later than 6 weeks after the last ravulizumab dose.
  • No adjustment in the elderly, mild or moderate renal impairment (eGFR 30 to <90 ml/min) or Child-Pugh A or B; no recommendation in severe renal impairment/dialysis; not recommended in Child-Pugh C.

Contraindications

  • Hypersensitivity to the active substance or any excipient.
  • Not vaccinated against Neisseria meningitidis and Streptococcus pneumoniae, unless the risk of delaying treatment outweighs the infection risk.
  • Unresolved infection with encapsulated bacteria (including Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae type B) at treatment initiation.

Important warnings

  • Serious, life-threatening or fatal infections with encapsulated bacteria may occur; vaccinate at least 2 weeks before the first dose, otherwise give antibacterial prophylaxis until 2 weeks after vaccination.
  • Monitor patients for early signs of serious infection and evaluate and treat immediately if suspected.
  • In PNH, discontinue only if clinically indicated and monitor for haemolysis afterwards (see the full text).

Side effects

  • PNH: most common upper respiratory tract infection (18.9%), headache (18.3%) and diarrhoea (11.0%); most common serious reaction urinary tract infection (1.2%).
  • C3G: most common upper respiratory tract infection (12.9%); most common serious reaction pneumococcal infection (1%).
  • Other common reactions: bronchitis, decreased platelet count, dizziness, abdominal pain, nausea.

Interactions

  • Strong inducers of CYP2C8, UGT1A1, PgP, BCRP and OATP1B1/3 (e.g. rifampicin) are not recommended (reduced efficacy).
  • Iptacopan may induce CYP3A4: caution with sensitive narrow-therapeutic-index CYP3A4 substrates (e.g. carbamazepine, ciclosporin, fentanyl, sirolimus, tacrolimus).

Pregnancy and breastfeeding

  • Limited data; no harmful effects in animal studies. PNH and C3G in pregnancy carry maternal and foetal risks; see the full text.

Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

Common questions – ask Medelix

For patients

Package leaflet, verbatim

This product has no separate US patient information.

Authorisation and brand names by country

Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.

EU – centrally authorised (all member states)

  • FabhaltaNovartis Europharm Limited · EMEA/H/C/005764

Source: European Medicines Agency (EMA), product information

Brand names in 3 more European countries
Iceland
Fabhalta
Liechtenstein
Fabhalta
Norway
Fabhalta

EMA – Article 57 product data (EU/EEA), 2026-10-29

Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.

Another question about this substance?

Medelix answers clinical questions from guidelines, studies and product information – every claim with its source.

Sources

  1. European Medicines Agency. Fabhalta – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/fabhalta-epar-product-information_en.pdf
  2. Wikidata (CC0): ATC L04A.

Editorial: Medelix editorial team · last updated 2026-10-28 · How these pages are made · Report an error

Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.