MITAPIVAT TAB
Clinical Criteria Summary
Indication & Patient Selection
- Hemolytic anemia due to pyruvate kinase (PK) deficiency in adults
- Requires at least one missense variant of the PKLR gene
- Excludes patients homozygous for PKLR mutation or with only non-missense mutations in PKLR gene
Dosage & Administration
- Starting dose: 5 mg twice daily for weeks 1-4
- Titrate to 20 mg twice daily if insufficient increase in hemoglobin
- Further titrate to maximum dose of 50 mg twice daily if hemoglobin is not increased and transfusion requirements are not reduced
- Available as 5 mg, 20 mg, and 50 mg oral tablets
- Dose taper packs available; abrupt cessation is not recommended
Safety & Monitoring Requirements
- Avoid abrupt discontinuation to minimize risk of acute hemolysis; gradual dose reduction recommended when possible
- Avoid concomitant use with strong CYP3A4 inhibitors or inducers
- Dose adjustment recommended if co-administration with moderate CYP3A inducer or inhibitor cannot be avoided
- Monitor for loss of therapeutic effect of drugs metabolized by CYP3A, CYP2B6/2C8/2C9/2C19 enzymes (mitapivat is an inducer of these enzymes)
Clinical Context & Utilization Restrictions
- Indicated for symptomatic anemia due to PK deficiency where standard supportive care includes transfusions and splenectomy
- No other drug therapies are approved for congenital hemolytic anemia due to PK deficiency
- Rare genetic condition; use within VA setting expected to be very infrequent