Jan. 03 2024, Agios announces Phase 3 ENERGIZE study of PYRUKYND (Mitapivat) met primary endpoint and both key secondary endpoints in adults with non-transfusion-dependent(NTD) alpha- or beta-thalassemia. The positive data showed Mitapivat’s potential to become the first oral therapy approved for NTD thalassemia.
Mitapivat is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the US, and for the treatment of PK deficiency in adult patients in the EU. By activating and stabilizing mutant pyruvate kinase, Mitapivat targets the underlying enzymatic defect that causes chronic hemolysis and ineffective erythropoiesis in order to restore enzyme activity.