Agios Pharmaceuticals Inc. (AGIO) is seeking to expand the label of PYRUKYND for use in the treatment of thalassemia, with a decision due on September 7, 2025.Thalassemia is an inherited blood disorder caused by mutations in either alpha- or beta-globin genes. This leads to a reduction in the production of functional hemoglobin, the protein in red blood cells that carries oxygen. There are two main types of thalassemia: Alpha thalassemia and Beta thalassemia. People with alpha- and beta-thalassemia experience a variety of health problems, like chronic anemia, debilitating fatigue, jaundice, facial bone deformities, delayed growth and development, and abdominal swelling, among others. If approved, PYRUKYND could be the first and only approved therapy indicated to treat all subtypes of thalassemia. PYRUKYND is already approved in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. The drug generated $12.5 million in net revenue for the second quarter of 2025, compared to $8.6 million in the second quarter of 2024.AGIO closed Wednesday’s (Aug.27, 2025) trading at $38.80, up 1.57%.