Beyond Gene Therapy: Pyruvate Kinase Activators (Agios, Novo Nordisk) Offer a New Route to Address Hemoglobin, VOCs, and Transfusion Burden in Sickle Cell Disease

The US sickle cell disease (SCD) market is evolving with increased interest in pyruvate kinase (PK) activators like mitapivat (Agios) and etavopivat (Novo Nordisk). These agents aim to improve red blood cell energy metabolism, potentially enhancing hemoglobin levels and reducing vaso-occlusive crises. Hematologists see promise in PK activators for both transfusion-dependent and non-transfusion-dependent patients, with about 40% of their SCD patients being suitable candidates. However, long-term efficacy and safety data are crucial for their integration into treatment regimens. The findings are part of Spherix Global Insights' Market Dynamix™ service, which analyzes specialty market dynamics.