Agios Pharmaceuticals, Inc., a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, announced that the U.S. Food and Drug Administration (FDA) has approved Pyrukynd (mitapivat) in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, lifelong hemolytic anemia. Pyrukynd is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease.

Hanny Al-Samkari, M.D., hematologist and clinical investigator at the Mass General Cancer Center and Harvard Medical School, and an investigator in these pivotal Phase 3 studies, said:

The successful ACTIVATE and ACTIVATE-T studies demonstrate the impact of mitapivat in significantly improving hemolysis and anemia in PK deficiency. The FDA approval of mitapivat, a targeted agent and first disease-modifying medication in PK deficiency, is an encouraging step forward for these patients that addresses a significant unmet need.

Pyrukynd is expected to be available in the U.S. approximately two weeks after approval. Pyrukynd was reviewed by the FDA under Priority Review and was previously granted orphan drug designation. Pyrukynd is also under review by the European Medicines Agency (EMA) as a potential treatment for adults with PK deficiency, and Agios expects a regulatory decision in the EU by the end of 2022.