Agios Gains FDA Approval for Anemia Drug Pyrukynd

February 22, 2022

The FDA has approved Agios Pharmaceuticals’ Pyrukynd (mitapivat), the first disease-specific treatment for hemolytic anemia in adults with pyruvate kinase deficiency.

The approval was supported by two phase 3 trials in which Pyrukynd was shown to increase hemoglobin in nonregularly transfused patients and reduce transfusion burden for regularly transfused patients.

Pyrukynd is also being reviewed by the European Medicines Agency for treating adults with pyruvate kinase deficiency, and a regulatory decision is expected by the end of 2022.

Agios also plans to launch two pivotal studies assessing Pyrukynd in children with pyruvate kinase deficiency later this year.

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