Agios’ PYRUKYND® (mitapivat) Receives Positive CHMP Opinion For Treatment Of Adults With Thalassemia

Agios Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company focused on delivering innovative therapies 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 a new indication for PYRUKYND® (mitapivat). PYRUKYND, an oral pyruvate kinase (PK) activator, is proposed for use in adults to treat anemia associated with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.

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

Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D at Agios, emphasized that the CHMP’s opinion represents an important milestone for the thalassemia community in Europe. She noted that thalassemia is a debilitating condition that places a significant burden on patients and their families, with few therapeutic options currently available. PYRUKYND represents a promising new medicine to address this unmet need, and the company looks forward to the potential of making it accessible 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. Agios will support the European commercial launch of PYRUKYND in thalassemia, pending regulatory approval. Avanzanite, headquartered in Amsterdam, is a commercial-stage specialty pharmaceutical company focused on delivering rare disease medicines to patients throughout Europe.

PYRUKYND is already approved in Saudi Arabia for the treatment of adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Additional regulatory applications are under review in the United States, with a Prescription Drug User Fee Act (PDUFA) target 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 United States and for the treatment of PK deficiency in adult patients in Europe.