Difference between revisions of "Mitapivat (Pyrukynd)"
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Pyruvate kinase activator | Pyruvate kinase activator | ||
| − | == | + | ==Diseases for which it is used== |
| − | ===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/study/NCT03692052 NCT03692052] | #'''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 NCT03692052] | ||
| − | ===Pyruvate kinase deficiency=== | + | ===[[Pyruvate kinase deficiency]]=== |
| − | |||
==History of changes in FDA indication== | ==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) | + | *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 deficiency|pyruvate kinase (PK) deficiency]]. ''(Based on ACTIVATE & ACTIVATE-T) |
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==Also known as== | ==Also known as== | ||
*'''Code name:''' AG-348 | *'''Code name:''' AG-348 | ||
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[[Category:Oral medications]] | [[Category:Oral medications]] | ||
[[Category:Pyruvate kinase activator]] | [[Category:Pyruvate kinase activator]] | ||
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| + | [[Category:Beta thalassemia medications]] | ||
| + | [[Category:Pyruvate kinase deficiency medications]] | ||
[[Category:FDA approved in 2022]] | [[Category:FDA approved in 2022]] | ||
Latest revision as of 13:25, 31 March 2024
Mechanism of action
Pyruvate kinase activator
Diseases for which it is used
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 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