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    Pyrukynd®

    Drug - Pyrukynd® (mitapivat) [Agios Pharmceuticals Inc.]

    October 2022

    Therapeutic area - Pyruvate kinase deficiency

    Initial approval criteria

    • Patient is at least 18 years of age AND
    • Patient has a confirmed diagnosis of pyruvate kinase deficiency (PKD) as defined by the documented presence of at least 2 variant alleles in the PKLR gene, of which at least 1 was a missense variant or PK enzyme deficiency AND
    • Patient is NOT homozygous for the c.1436G>A (p.R479H) variant AND
    • Patient has a baseline serum hemoglobin level < 10 g/dL or required more than 6 transfusions in the prior year AND
    • Other causes of hemolytic anemia have been ruled out (e.g., immune hemolysis, other enzyme deficiencies, vitamin/mineral deficiencies) AND
    • Patient does not have moderate or severe hepatic impairment AND
    • Patient will avoid concomitant therapy with all of the following:
      • Coadministration with strong CYP3A4 inhibitors (e.g., nefazodone, itraconazole) AND
      • Coadministration with moderate CYP3A4 inhibitors (e.g., ciprofloxacin, verapamil); if therapy is unavoidable, the patient will be monitored closely for adverse reaction and/or dose modifications will be implemented AND
      • Coadministration with strong CYP3A inducers (e.g., rifampin, carbamazepine, St. John’s wort) AND
      • Coadministration with moderate CYP3A inducers (e.g., bosentan, modafinil, nafcillin); if therapy is unavoidable, the patient will be monitored closely for adverse reaction and/or dose modifications will be implemented AND
    • Prescriber will advise patients currently on hormonal contraceptives to use an alternative non-hormonal contraceptive method or add a barrier method of contraception during treatment
    • Initial approval is for 6 months

    Renewal criteria

    • Patient must continue to meet the above criteria AND
    • Patient has shown a beneficial response to therapy compared to pre-treatment baseline in 1 or more of the following:
      • Hemoglobin (Hb) response (defined as a ≥ 1.5 g/dL increase in hemoglobin level without transfusion over a 4-week or longer time period) OR
      • Transfusion reduction response (defined as a ≥ 33% reduction in the number of red blood cell [RBC] units transfused compared to historical transfusion burden) OR
      • Patient had an increase in Hb and/or decrease in transfusion requirement, to a lesser extent than the above, AND also had an improvement in the signs and symptoms (e.g., fatigue, jaundice, shortness of breath) and/or markers of hemolysis (e.g., indirect bilirubin, reticulocyte count, LDH, haptoglobin)
    • Renewal approval is for 6 months

    Quantity limits

    • 5 mg, 20 mg, 50 mg tablets: 60 tablets/30 days 

    Questions?

    MHCP Provider Resource Center 651-431-2700 or 800-366-5411

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