IPTACOPAN CAP,ORAL

Clinical Criteria Summary

Active infection with Neisseria meningitidis, Neisseria gonorrhoeae, Haemophilus influenzae, or Streptococcus pneumoniae

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)

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

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

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

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