NEJM: Efficacy of Mitapivat in the Treatment of Pyruvate Kinase Deficiency (ACTIVATE Trial)

Pyruvate kinase deficiency is a rare, hereditary, chronic disease associated with hemolytic anemia

In this global, randomized, placebo-controlled Phase 3 clinical trial, researchers evaluated the efficacy and safety of Mitapivat in adult patients with pyruvate kinase deficiency who were not receiving normal red blood cell transfusions

The Phase 3 ACTIVATE trial of mitapivat met its primary endpoint

Jaundice (LS mean difference PYRUKYND minus placebo: -0.

The most common adverse events were nausea (7 patients [18%] in the Mitapivat group and 9 patients [23%] in the placebo group) and headache (6 patients [15%] and 13 patients [33%], respectively)

The most common adverse reactions in patients with PK deficiency included laboratory abnormalities (≥10%) were decreased estrone (males), increased uric acid, back pain, decreased estradiol (males), and arthralgia

CONCLUSIONS: In patients with pyruvate kinase deficiency, Mitapivat significantly increased hemoglobin levels, reduced hemolysis, and improved patient-reported outcomes .