Agios announces Pyrukynd approval in the EU

Cambridge, USA-based drug developer Agios Pharmaceuticals (Nasdaq: AGIO) announced that the European Commission has granted marketing authorization for Pyrukynd (mitapivat), an oral pyruvate kinase (PK) activator, in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, with an orphan medicinal product designation.

Dr Raffaella Origa, professor of pediatrics at University of Cagliari, Italy and president of the Italian Society of Thalassaemia and Hemoglobinopathies (SITE), commented: “The approval of Pyrukynd in the EU represents an important step forward, introducing a new oral treatment option regardless of genotype or transfusion burden, with the potential to address key aspects of the disease, including reducing transfusion burden and improving patient outcomes.”