[3] Mitapivat was approved for medical use in the United States in February 2022,[1][3][4][5] and in the European Union in November 2022.

[8][9] The FDA approved mitapivat based on evidence from two clinical trials of 107 participants with pyruvate kinase deficiency.

[3] Trial 1 (NCT03548220) of 80 adults with pyruvate kinase deficiency who did not receive regular blood transfusions and trial 2 (NCT03559699) of 27 adults with pyruvate kinase deficiency who received regular blood transfusions.

[3] In trial 1, participants were randomly assigned to receive either mitapivat or a matched placebo tablet for an average duration of about 24 weeks.

[10] The applicant for this medicinal product is Agios Netherlands B.V.[10] Mitapivat was approved for medical use in the European Union in November 2022.