Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the European Commission (EC) has granted marketing authorization for PYRUKYND® (mitapivat), an oral pyruvate kinase (PK) activator, in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, with an orphan medicinal product designation. With this decision, PYRUKYND becomes the only medicine approved in all European Union (EU) member states for this broad patient population.

"Thalassemia imposes a profound daily burden on patients, which is compounded by a lack of accessible treatment options for all forms of the disease," said Maria Domenica Cappellini, M.D., Professor of Internal Medicine, University of Milan, Italy, and an investigator in the PYRUKYND thalassemia Phase 3 clinical program. "The Phase 3 results demonstrate the potential for PYRUKYND to address this burden and improve outcomes for those in need, regardless of their genotype or transfusion status. This approval is a major advancement that can help thousands of patients across the EU manage this debilitating and life-threatening disease."

The EC's decision is based on the results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively.

"The EC approval of PYRUKYND is an important achievement, marking meaningful progress for patients living with thalassemia," said Brian Goff, Chief Executive Officer, Agios. "Securing approvals across four key markets – the U.S., Saudi Arabia, United Arab Emirates, and now the EU – validates the transformative potential of this novel medicine. We look forward to continuing our partnership with Avanzanite to bring PYRUKYND to thalassemia patients across the EU, furthering our commitment to advancing innovation for the global community."

In 2025, Agios entered into an exclusive agreement with Avanzanite Bioscience B.V. (Avanzanite) for the commercialization and distribution of PYRUKYND across the European Economic Area, the United Kingdom, and Switzerland. Headquartered in Amsterdam, Avanzanite is a commercial-stage specialty pharmaceutical company dedicated to bringing rare disease medicines to patients across Europe.

"The global thalassemia community has long awaited new, convenient, safe, and effective treatment options that help alleviate the debilitating symptoms of this disease," said Androulla Eleftheriou, Ph.D., Executive Director, Thalassaemia International Federation. "The EC's decision is a welcome step forward, introducing patients in the EU to an oral medicine with demonstrated therapeutic benefits. With four regulatory approvals in geographic areas significantly affected by thalassemia, this milestone reflects growing momentum to improve outcomes for patients worldwide."

Mitapivat is now approved for adults with thalassemia in the EU, Saudi Arabia, and United Arab Emirates under the brand name PYRUKYND, as well as in the U.S. under the brand name AQVESME™ (mitapivat). The medicine is also approved as PYRUKYND for adults with PK deficiency in both the U.S. and in Europe.