Agios Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency.
Agios Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s first-in-class pyruvate kinase-R (PKR) activator mitapivat for the treatment of patients with thalassemia.