Agios Drug Wins FDA Approval

Cambridge-based Agios Pharmaceuticals has announced that the FDA has approved Pyrukynd in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, chronic hemolytic anemia. Pyrukynd is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease. The drug was reviewed by the FDA under Priority Review and was previously granted orphan drug designation. Pyrukynd is also under review by the European Medicines Agency (EMA) as a potential treatment for adults with PK deficiency, and Agios expects a regulatory decision in the EU by the end of 2022.

A year ago, Agios sold its oncology portfolio to Servier for roughly $2.0 billion, including $1.8 billion in upfront cash. At that time, CEO Jackie Fouse explained the move as the result of a strategic review and reflected the progress Agios had made “understanding and harnessing the science and promise of PK activation.” She added “With mitapivat [Pyrukynd] poised to become a new potential treatment option for patients with pyruvate kinase (PK) deficiency, thalassemia and sickle cell disease and with a rich pipeline based on our pioneering leadership in PK activation and cellular metabolism, Agios’ near- and long-term future is filled with significant value-generating catalysts.” With FDA approval for Pyrukynd in hand, Agios has taken a significant step toward fulfilling its predictions.

Pyruvate kinase deficiency is a rare, inherited disease that presents as chronic hemolytic anemia. PK deficiency is associated with serious complications, including gallstones, pulmonary hypertension, extramedullary hematopoiesis, osteoporosis and iron overload.. PK deficiency can also cause quality of life problems, including challenges with work and school activities, social life and emotional health. Current management strategies for PK deficiency, including red blood cell transfusions and splenectomy, are associated with both short- and long-term risks.  (Source: Agios Website, 21 December, 2021 and 17 February, 2022)