Difference between revisions of "Mitapivat (Pyrukynd)"
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m (Jwarner moved page Mitapivat (AG-348) to Mitapivat (Pyrukynd): FDA approval) |
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==Preliminary data== | ==Preliminary data== | ||
===Beta thalassemia=== | ===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. [https://doi.org/10.1016/s0140-6736(22)01337-x link to original article] [https://pubmed.ncbi.nlm.nih.gov/35964609/ PubMed] [https://clinicaltrials.gov/ | + | #'''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. [https://doi.org/10.1016/s0140-6736(22)01337-x link to original article] [https://pubmed.ncbi.nlm.nih.gov/35964609/ PubMed] [https://clinicaltrials.gov/study/NCT03692052 Clinical Trial Registry] |
===Pyruvate kinase deficiency=== | ===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. [https://doi.org/10.1056/nejmoa1902678 link to original article] [https://pubmed.ncbi.nlm.nih.gov/31483964/ PubMed] [https://clinicaltrials.gov/ | + | #'''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. [https://doi.org/10.1056/nejmoa1902678 link to original article] [https://pubmed.ncbi.nlm.nih.gov/31483964/ PubMed] [https://clinicaltrials.gov/study/NCT02476916 Clinical Trial Registry] |
==History of changes in FDA indication== | ==History of changes in FDA indication== | ||
Revision as of 01:31, 25 June 2023
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
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