Drug updated on 4/18/2024

Dosage FormTablet (oral; 5 mg, 20 mg, 50 mg)
Drug ClassPyruvate kinase activators
Ongoing and
Completed Studies


  • Indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

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  • Mitapivat (Pyrukynd) is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, showing effectiveness in both transfusion-dependent and non-transfusion-dependent populations.
  • Two randomized controlled studies were conducted to gather information about this drug; one study focused on PK deficient patients not receiving regular red-cell transfusions, while the other targeted those who were regularly receiving transfusions.
  • In the ACTIVATE study involving 80 participants, mitapivat significantly outperformed a placebo with 40% of patients showing a sustained increase in hemoglobin levels compared to none from the placebo group.
  • The second study, ACTIVATE-T, involved 27 participants all requiring regular blood transfusions prior to treatment; it found that 37% experienced at least a third reduction in their need for such procedures after being administered mitapivat.
  • Regarding safety concerns raised by these studies: adverse events of grade three or higher occurred among a quarter of subjects taking mitapivat versus only thirteen percent from those given placebos according to results from ACTIVATE. Meanwhile, increases in alanine aminotransferase and headaches each affected thirty-seven percent of participants during ACTIVATE-T, but no deaths related directly to treatments were reported.
  • While direct comparisons between mitapivat and other therapies are lacking within these studies due to its novelty as a disease-modifying option against PK deficiency, which was previously managed primarily through supportive care like blood transfers, its potential superiority can be inferred based on its demonstrated capabilities highlighted across both studies.

Product Monograph / Prescribing Information

Document TitleYearSource
Pyrukynd (mitapivat) Prescribing Information. 2022Agios Pharmaceuticals, Inc., Cambridge, MA

Randomized Controlled Trials

Sex Distribution:




Source:New England Journal of Medicine