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iptacopan (Fabhalta)
Indications:
- treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
Contraindications:
- not recommended with severe renal impairment or severe hepatic impairment
Dosage:
- 200 mg PO BID with or without food.
Capsules: 200 mg (3)
Monitor:
- monitor for hemolysis after discontinuation
Pharmacokinetics:
- peak plasma concentrations in ~2 hours
- 1/2life of 25 hours
- steady state level reached in 5 days
- clearance is 8 liters/hour at steady state concentration
- absorption not affected by high fat meals
- protein binding 75-93%
- volume of distribution 288 liters
- metabolism by CYP2C8 via N-dealkylation, O-deethylation, oxidation, & dehydrogenation with phase 2 metabolism through glucuronidation by UGT1A1, UGT1A3, & UGT1A8
- iptacopan is major component in plasma (83%)
- no active metabolites
Adverse effects:
- > 10%
- headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection*, viral infection, nausea and rash
- no effect on QTc interval
* serious & life-threatening infections caused by encapsulated bacteria, including
- Streptococcus pneumoniae, Neisseria meningitidis, & Haemophilus influenzae type B
Drug interactions:
- CYP2C8 inducers (rifampin) reduces plasma levels of iptacopan
Mechanism of action:
- complement factor B inhibitor
- regulates cleavage of complement C3, generation of downstream effectors, & amplification of the terminal pathway.
Notes:
- available only through a restricted program under a Risk Evaluation & Mitigation Strategy (REMS) called FABHALTA REMS.
General
hematologic agent
Database Correlations
PUBCHEM correlations
References
- HIGHLIGHTS OF PRESCRIBING INFORMATION
Fabhalta (iptacopan) capsules, for oral use
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/218276s000lbl.pdf