Agios Is Closer to FDA Decision for PYRUKYND in Thalassemia, and Plans Expansion

Agios Pharmaceuticals is on track to deliver a major milestone in rare disease therapy, with the U.S. FDA decision for PYRUKYND® (mitapivat) in thalassemia expected by September 7, 2025. If approved, this oral pyruvate kinase activator could become the first disease-modifying treatment for non-transfusion-dependent thalassemia, addressing a critical unmet need. CEO Brian Goff emphasized Agios’ $1.4 billion cash reserves to support the launch and expansion into sickle cell disease (2026) and global markets. Recent Phase 3 data in PK deficiency and progress with tebapivat further solidify the company’s leadership in rare blood disorders. Dr. Sarah Gheuens, Agios’ CMO, noted, “PYRUKYND’s mechanism — improving red blood cell health — has transformative potential across thalassemia, SCD, and PK deficiency.”

At OxyDial, we recognize the urgency of innovative therapies for underserved communities, where thalassemia and SCD impose heavy burdens. PYRUKYND’s potential marks a turning point.