Agios' PYRUKYND® (mitapivat) Receives Positive CHMP Opinion for Adults with Thalassemia

Core Viewpoint - Agios Pharmaceuticals has received a positive opinion from the CHMP of the EMA for the new indication of PYRUKYND® (mitapivat) to treat anemia associated with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia in adults [1][2][3] Company Developments - The CHMP's opinion is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE-T and ENERGIZE Phase 3 trials [2] - Agios has entered into an exclusive agreement with Avanzanite Bioscience B.V. for the commercialization and distribution of PYRUKYND across the European Economic Area, the UK, and Switzerland [3] - PYRUKYND has already received approval in Saudi Arabia for treating adult patients with both types of thalassemia [4] Clinical Trials - The ENERGIZE trial involved 194 non-transfusion-dependent patients, with a primary endpoint of achieving a hemoglobin response defined as an increase of ≥1.0 g/dL [10] - The ENERGIZE-T trial included 258 transfusion-dependent patients, focusing on a transfusion reduction response defined as a ≥50% reduction in transfused RBC units [11] Disease Background - Thalassemia is a rare inherited blood disease affecting hemoglobin production, categorized into alpha-thalassemia and beta-thalassemia [6] - An estimated 18,000 to 23,000 individuals are living with thalassemia in the U.S. and the five largest European countries [8]