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Global Medical Affairs


Resources for Healthcare Providers

This site contains educational and scientific information relating to Agios investigational and approved medicines.

Scientific resources

PK Deficiency

Pyruvate kinase (PK) deficiency is a rare, hereditary, chronic hemolytic anemia caused by mutations in the PKLR gene encoding the enzyme pyruvate kinase, which is critical for maintaining red blood cell (RBC) energy levels and normal RBC life span. Defects in pyruvate kinase lead to premature destruction of erythrocytes which manifest clinically as anemia and serious complications including gallstones, pulmonary hypertension, thrombosis, osteopenia, osteoporosis, and iron overload. Currently, Agios is studying the long-term safety and efficacy of PK activation in adults with PK deficiency and has expanded the clinical trial program to study PK activation in pediatric patients with PK deficiency from 1-17 years of age.

PK Deficiency Resources for HCPs


Thalassemia is a diverse group of genetic disorders with a worldwide distribution that are characterized by reduced or absent production of hemoglobin. Imbalanced globin chain production that occurs in thalassemic red blood cells leads to ineffective erythropoiesis, hemolysis, and dysregulated iron homeostasis, resulting in the development of anemia and other clinical complications. Although important advancements in the management of thalassemia have been made over the last few decades, significant unmet needs remain that are not addressed by current approaches. Agios is studying how PK activation could potentially benefit people across the full range of thalassemia types, including both α- and β-thalassemia. PK activation increases adenosine triphosphate (ATP) and enhances the energy metabolism of the red blood cell (RBC), which may lead to improved membrane integrity and RBC health. Currently, Agios is studying PK activation in adults with non-transfusion-dependent and transfusion-dependent thalassemia in ongoing clinical trials to assess effects on both anemia and transfusion burden.

Thalassemia Resources for HCPs

Sickle Cell Disease

Sickle cell disease (SCD), a monogenetic disease of hemoglobin, affects millions across the globe. The resulting sickled red blood cell (RBC) leads to a host of complications including anemia, hemolysis, and episodes of acute pain, among others. People with SCD continue to have needs not met by current therapies. At Agios, we are studying how PK activation could potentially benefit people with SCD. PK activation reduces 2,3-diphosphoglycerate (2,3-DPG) levels in RBCs which increases hemoglobin oxygen affinity, reducing hemoglobin S polymerization and may inhibit the sickling process. At the same time, increasing adenosine triphosphate (ATP) enhances the energy metabolism of the RBC which may lead to improved membrane integrity and RBC health. Currently, Agios is studying PK activation in adults with SCD in ongoing clinical trials to assess both the effect on anemia and vaso-occlusive events.

Sickle Cell Disease Resources for HCPs


Investigator-Sponsored Trials

Agios supports Investigator Sponsored Trials (ISTs), which are independent, investigator-initiated research designed to advance scientific knowledge of disease states, patient populations and medical treatment in alignment with our clinical and nonclinical areas of interest.

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