Drug updated on 4/18/2024
| Dosage Form | Tablet (oral; 5 mg, 20 mg, 50 mg) |
| Drug Class | Pyruvate kinase activators |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
Summary
- 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 Title | Year | Source |
|---|---|---|
| Pyrukynd (mitapivat) Prescribing Information. | 2022 | Agios Pharmaceuticals, Inc., Cambridge, MA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Mitapivat versus placebo for pyruvate kinase deficiency. | 80Subjects F: 60% M: 40% | 2022 | New England Journal of Medicine |
| Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): A multicentre, open-label, single-arm, phase 3 trial. | 27Subjects F: 74.1% M: 25.9% | 2022 | The Lancet |
Document Title
Sex Distribution:
F:60%
M:40%
80Subjects
Year:
2022
Source:New England Journal of Medicine
Sex Distribution:
F:74.1%
M:25.9%
27Subjects
Year:
2022
Source:The Lancet