Agios Pharma gets US FDA nod for mitapivat to treat hemolytic anemia in adults with pyruvate kinase deficiency
Agios Pharmaceuticals, Inc, a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, announced that the US health regulator has approved PYRUKYND (mitapivat) in the US 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.
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