Mitapivat (Pyrukynd)
Revision as of 15:33, 31 May 2023 by Warner-admin (talk | contribs) (Text replacement - "(NCT[0-9]{8})" to "[https://clinicaltrials.gov/ct2/show/$1 Clinical Trial Registry]")
Mechanism of action
Pyruvate kinase activator
Preliminary data
Beta thalassemia
- 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 Clinical Trial Registry
Pyruvate kinase deficiency
- AG348-C-003: Grace RF, Rose C, Layton DM, Galactéros F, Barcellini W, Morton DH, van Beers EJ, Yaish H, Ravindranath Y, Kuo KHM, Sheth S, Kwiatkowski JL, Barbier AJ, Bodie S, Silver B, Hua L, Kung C, Hawkins P, Jouvin MH, Bowden C, Glader B. Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. N Engl J Med. 2019 Sep 5;381(10):933-944. link to original article PubMed Clinical Trial Registry
Also known as
- Code name: AG-348