Agios Pharmaceuticals Inc. (NASDAQ: AGIO) announced that the European Commission has granted marketing authorization for PYRUKYND (mitapivat) for treating anemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.

The oral pyruvate kinase activator received orphan medicinal product designation and becomes the only medicine approved across all European Union member states for this patient population. The approval follows successful Phase 3 clinical trials called ENERGIZE and ENERGIZE-T, which evaluated the drug's efficacy in both transfusion-dependent and non-transfusion-dependent thalassemia patients.

Mitapivat is now approved for thalassemia treatment in four markets: the United States (under brand name AQVESME), Saudi Arabia, United Arab Emirates, and the EU (all under brand name PYRUKYND). The drug is also approved for pyruvate kinase deficiency treatment in the U.S. and Europe under the PYRUKYND brand.

Avanzanite Bioscience B.V., an Amsterdam-based specialty pharmaceutical company, will handle distribution and commercialization of PYRUKYND across Europe under an exclusive agreement with Agios established in 2025. The partnership covers the European Economic Area, United Kingdom, and Switzerland.

Thalassemia affects hemoglobin production in red blood cells, leading to anemia and serious complications. An estimated 18,000 to 23,000 children and adults live with the condition in the U.S. and five largest European countries combined. The disease requires some patients to receive regular blood transfusions while others need them intermittently.

The ENERGIZE trial studied 194 non-transfusion-dependent patients, while ENERGIZE-T examined 258 transfusion-dependent patients. Both trials compared mitapivat 100 mg twice daily against placebo in randomized, double-blind studies.