|Other names||AG-348, Mitapivat sulfate (USAN US)|
|Drug class||Pyruvate kinase activator|
|Main uses||Hemolytic anemia in pyruvate kinase deficiency|
|Side effects||Trouble sleeping, back pain, nausea, joint pain|
|Chemical and physical data|
|Molar mass||450.56 g·mol−1|
|3D model (JSmol)|
Mitapivat was approved for medical use in the United States and Europe in 2022. In the United States 4 weeks of treatment costs about 27,000 USD as of 2022. It is not commercially available in the United Kingdom as of 2022.
Mechanism of action
Mitapivat binds to and activates pyruvate kinase, thereby enhancing glycolytic pathway activity, improving adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG) levels. Mutations in pyruvate kinase cause deficiency in pyruvate kinase which prevents adequate red blood cell (RBC) glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the pyruvate kinase product ATP.
It is taken as the sulfate hydrate salt.
Society and culture
On 15 September 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Pyrukynd, intended for the treatment of an inherited condition called pyruvate kinase deficiency. The applicant for this medicinal product is Agios Netherlands B.V. Mitapivat was approved for medical use in the European Union in November 2022.
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- Gormley N. "Pyrukynd (mitapivat) tablets NDA approval" (PDF). Center for Drug Evaluation and Research. Letter to Christina Baladi (Agios Pharmaceuticals, Inc.). U.S. Food and Drug Administration.
- "Mitapivat (Code C157039)". NCI Thesaurus. 31 January 2022. Archived from the original on 20 February 2022. Retrieved 19 February 2022. This article incorporates text from this source, which is in the public domain.
- "PK-R allosteric activator AG-348". NCI Drug Dictionary. National Cancer Institute. Archived from the original on 10 August 2019. Retrieved 19 February 2022. This article incorporates text from this source, which is in the public domain.
- "Pyrukynd: Pending EC decision". European Medicines Agency (EMA). 15 September 2022. Archived from the original on 19 September 2022. Retrieved 18 September 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- World Health Organization (2017). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 78". WHO Drug Information. 31 (3): 539. hdl:10665/330961.
- "Mitapivat sulfate". Drug Information Portal. U.S. National Library of Medicine. Archived from the original on 2022-08-15. Retrieved 2022-12-09.
- Clinical trial number NCT03548220 for "A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)" at ClinicalTrials.gov
- Clinical trial number NCT03559699 for "A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)" at ClinicalTrials.gov