Agios Pharmaceuticals receives positive CHMP opinion for PYRUKYND

Agios Pharmaceuticals (AGIO) announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion for the new indication for PYRUKYND, an oral pyruvate kinase activator, in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The CHMP’s opinion is based on the results from the global, randomized, double-blind, placebo-controlled ENERGIZE-T and ENERGIZE Phase 3 trials in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, respectively. The European Commission will now review the CHMP’s opinion, with the final decision expected by early 2026.