Mitapivat (Pyrukynd)

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Mechanism of action

Pyruvate kinase activator

Diseases for which it is used

Beta thalassemia

  1. AG348-C-010: Kuo KHM, Layton DM, Lal A, Al-Samkari H, Bhatia J, Kosinski PA, Tong B, Lynch M, Uhlig K, Vichinsky EP. Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study. Lancet. 2022 Aug 13;400(10351):493-501. link to original article PubMed NCT03692052

Pyruvate kinase deficiency

History of changes in FDA indication

  • 2022-02-17: Initial approval to treat hemolytic anemia (a disorder in which red blood cells are destroyed faster than they can be made) in adults with pyruvate kinase (PK) deficiency. (Based on ACTIVATE & ACTIVATE-T)

Also known as

  • Code name: AG-348
  • Brand name: Pyrukynd