VA Criteria for Use Formulary Advisor Reference
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IPTACOPAN CAP,ORAL

Generic Name
IPTACOPAN CAP
Brand Names
FABHALTA
Drug Class
BLOOD PRODUCTS,OTHER
Formulary Status
Non-Formulary Requires prior authorization
Copay Tier
3
Last Updated
December 1, 2024

Clinical Criteria Summary

Document 634: Pegcetacoplan Iptacopan Danicopan for Paroxysmal Nocturnal Hemoglobinuria Criteria

⇄ This criteria document covers 3 drugs across 3 drug classes.
See all drugs in this document
  • IPTACOPAN CAP,ORAL
  • PEGCETACOPLAN INJ,SOLN
  • PEGCETACOPLAN INJ,SOLN

Criteria for Paroxysmal Nocturnal Hemoglobinuria (PNH) - Alternative Pathway Complement Inhibitors (Danicopan, Iptacopan, Pegcetacoplan)

  • Exclusion Criteria
  • Active infection with Neisseria meningitidis, Neisseria gonorrhoeae, Haemophilus influenzae, or Streptococcus pneumoniae
  • Inclusion Criteria
  • Must be prescribed by a REMS registered VA or VA Community Care hematologist, oncologist, immunologist or genetic specialist
  • Laboratory-confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), as evidenced by detectable GPI-deficient hematopoietic clones (Type III PNH red blood cells (RBC)) via Flow Cytometry
  • Evidence of clinically significant hemolysis (e.g., Hemoglobin <10g/dL) despite 6 months of stable therapy with anti-C5 inhibitor (e.g., ravulizumab or eculizumab)
  • Complete or update vaccination for encapsulated bacteria, including Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks prior to the first dose of therapy according to current Advisory Committee on Immunization Practices (ACIP)
  • Note: All criteria listed above apply collectively to Danicopan, Iptacopan, and Pegcetacoplan for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Document 646: MON Iptacopan FABHALTA Monograph Sept 2024

Indication & Population

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Adult patients with confirmed PNH and hemoglobin <10g/dL

Dosage & Administration

  • 200mg oral capsules administered twice daily

Contraindications

  • Hypersensitivity to iptacopan
  • Unresolved serious infection caused by encapsulated bacteria (Streptococcus pneumoniae, Neisseria meningitidis, or H. influenzae type B)

Warnings & Precautions

  • Risk of serious infections caused by encapsulated bacteria
  • Use not recommended in severe renal impairment (eGFR<30mL/min) or severe hepatic impairment (Child-Pugh class C)
  • Insufficient data to make recommendation regarding risk of birth defects in pregnancy
  • Breastfeeding cessation recommended during treatment and for 5 days after final dose

Drug Interactions

  • Strong CYP2C8 inducers (e.g., rifampin) or inhibitors (e.g., gemfibrozil) can significantly decrease or increase iptacopan exposure

Monitoring & Pre-treatment Requirements

  • Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose, unless risks of delaying therapy outweigh risk of developing a serious infection
  • Monitor for hemolysis after cessation of therapy

Source Documents

Document 634: Pegcetacoplan Iptacopan Danicopan for Paroxysmal Nocturnal Hemoglobinuria Criteria Document 646: MON Iptacopan FABHALTA Monograph Sept 2024