CHMP recommends Pyrukynd for the treatment of pyruvate kinase deficiency in adult patients
Agios Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending the granting of a marketing authorization for Pyrukynd (mitapivat) for the treatment of pyruvate kinase (PK) deficiency in adult patients
PK deficiency is a rare, debilitating, lifelong hemolytic anemia. Pyrukynd is a first-in-class, oral PK activator that was recently approved by the FDA. If approved by the European Commission (EC), Pyrukynd will be the first approved disease-modifying therapy for European patients with PK deficiency.
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