The U.S. Food and Drug Administration (FDA) has approved Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.
Alpha- and beta-thalassemia are blood disorders that reduce the body’s production of hemoglobin. Affected people have anemia, which can cause pale skin, weakness, fatigue, and more serious complications. Alpha-thalassemia typically results from deletions involving the HBA1 and HBA2 genes. Beta-thalassemia is caused by genetic changes in the HBB gene.
Mitapivat is an oral pyruvate kinase (PK) activator. It is the only medicine approved for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
The approval is based on results from the phase 3 global, randomized, double-blind, placebo-controlled ENERGIZE (NCT04770753) and ENERGIZE-T (NCT04770779) clinical trials. The trials enrolled a total of 452 patients and met all primary and secondary key efficacy endpoints. Mitapivat was observed to improve hemolytic anemia and quality-of-life measures compared to placebo. These included significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue.
The medication does come with a Black Box Warning. Five patients receiving mitapivat experienced adverse reactions that suggested hepatocellular injury, with two requiring hospitalization. These events occurred within the first six months of treatment and improved upon discontinuation. To mitigate this risk, mitapivat is only available through the Aqvesme Risk Evaluation and Mitigation Strategy program approved by the FDA.
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To learn more about thalassemia and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/