Agios Secures EU Marketing Authorization for PYRUKYND in Alpha and Beta Thalassemia
European Commission granted marketing authorization for PYRUKYND (mitapivat) as the only medicine approved for adults with transfusion-dependent and non-transfusion-dependent alpha- and beta-thalassemia across all EU states, following positive Phase 3 ENERGIZE and ENERGIZE-T results. Agios will partner with Avanzanite Bioscience to commercialize PYRUKYND in the European Economic Area, UK, Switzerland.
1. EU Marketing Authorization
European Commission has granted marketing authorization for PYRUKYND in adults with both transfusion-dependent and non-transfusion-dependent alpha- and beta-thalassemia. This orphan medicinal product designation makes PYRUKYND the only authorized treatment for this broad thalassemia population across all EU states.
2. Phase 3 Trial Evidence
Approval was based on global ENERGIZE and ENERGIZE-T Phase 3 trials, which enrolled 194 non-transfusion-dependent and 258 transfusion-dependent patients respectively. Both trials met primary endpoints with significant hemoglobin increases and transfusion reduction responses over placebo.
3. Commercial Partnership
In 2025 Agios entered into an exclusive distribution and commercialization agreement with Avanzanite Bioscience B.V. for the European Economic Area, United Kingdom, and Switzerland. Avanzanite will handle market launch, supply chain management, and physician outreach for PYRUKYND.
4. Global Approval Footprint
PYRUKYND is now approved in four key markets including the U.S. (AQVESME in thalassemia and PK deficiency) as well as Saudi Arabia and the United Arab Emirates. This expanded footprint underscores Agios's strategy to address rare blood disorders globally.