Summary

This AI-generated content is provided without warranty, with no liability accepted for reliance on it. Learn more.

• Pyrukynd (mitapivat) is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• Hemoglobin Response: In a global, phase 3, randomized, placebo-controlled trial involving adults with pyruvate kinase deficiency not receiving regular transfusions, 40% of patients in the mitapivat group achieved a hemoglobin response (≥1.5 g/dL increase from baseline), compared to 0% in the placebo group. The adjusted difference was 39.3 percentage points (95% CI, 24.1 to 54.6; P<0.001).
• Reduction in Transfusion Burden: In an open-label, single-arm, phase 3 trial (ACTIVATE-T) involving adults with pyruvate kinase deficiency receiving regular transfusions, 37% of participants experienced a ≥33% reduction in the number of RBC units transfused during the fixed-dose period, compared to their historical transfusion burden (95% CI, 19-58; P=0.0002).
• Nausea and headache were reported as common adverse events, with nausea occurring in 18% of the mitapivat group versus 23% in the placebo group, and headache occurring in 15% of the mitapivat group versus 33% in the placebo group. Adverse events included an increase in alanine aminotransferase (37%), headache (37%), increase in aspartate aminotransferase (19%), fatigue (19%), and nausea (19%).
• Severe adverse events (grade 3 or higher) were observed in 25% of patients in the mitapivat group compared to 13% in the placebo group. Two grade 3 treatment-related adverse events (joint swelling and increase in aspartate aminotransferase, each in 4%) and three serious treatment-emergent adverse events not related to the study treatment were also reported. No new safety signals were identified in patients receiving mitapivat.
• The reviewed studies focused on adults with pyruvate kinase deficiency, differentiating between those not receiving regular transfusions and those receiving regular transfusions. In the latter, the population was predominantly female (74%) and White (74%), with 11% Asian participants. No specific safety or effectiveness differences among these subgroups were reported.