Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases,announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the new indication 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.

The CHMP’s opinion is based on the results from the global, randomized, double-blind, placebo-controlled ENERGIZE-T and ENERGIZE Phase 3 trials in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, respectively. The European Commission will now review the CHMP’s opinion, with the final decision expected by early 2026.

“The CHMP’s positive opinion marks an important step forward for the thalassemia community in Europe,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “Thalassemia is a debilitating disease that places a profound burden on patients and families, with few, if any, therapeutic options currently available to help manage the condition. PYRUKYND represents a promising new medicine to address this urgent need, and we look forward to the potential of bringing it to patients across Europe.”

In June 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, and will support the European commercial launch of PYRUKYND in thalassemia, pending approval. Headquartered in Amsterdam, Avanzanite is a commercial-stage specialty pharmaceutical company dedicated to bringing rare disease medicines to patients across Europe.

PYRUKYND has received approval in Saudi Arabia for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Additional regulatory applications for PYRUKYND in thalassemia are under review by health authorities in the U.S., with a Prescription Drug User Fee Act (PDUFA) goal date of December 7, 2025, as well as in the United Arab Emirates.

PYRUKYND is also approved for the treatment of hemolytic anemia in adults with PK deficiency in the U.S. and for the treatment of PK deficiency in adult patients in Europe.