25/12/2025
Major Milestone for the Global Thalassaemia Community
The U.S. Food and Drug Administration (FDA) has approved ™ ( ), developed by Agios Pharmaceuticals, for the treatment of anaemia in adult with α- or β- — covering both transfusion-dependent (TDT) and non-transfusion-dependent (NTDT) forms of the disorder.
This marks the first-ever therapy indicated across both patient groups, representing a truly historic advance in the management of this complex hereditary .
AQVESME is an oral pyruvate kinase (PK) activator, shown in the global Phase III ENERGIZE and ENERGIZE-T clinical studies to deliver statistically significant and clinically meaningful improvements in and , as well as reductions in compared to placebo among 452 enrolled patients.
Brian Goff, Chief Executive Officer of Agios, stated: “Today is a landmark moment for the thalassemia community, bringing forward an innovative, disease-modifying oral medicine to address the urgent needs of people living with this devastating rare blood disorder.”
