米他匹伐：丙酮酸激酶缺乏症成人溶血性贫血的一种新型治疗方法。

Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency.

作者信息

De Surya K

机构信息

Department of Chemistry, Conju-Probe, San Diego, California, USA.

Bharath University, Chennai, Tamil Nadu, 600126, India.

出版信息

Curr Med Chem. 2024;31(6):683-687. doi: 10.2174/0929867330666230430002709.

DOI:10.2174/0929867330666230430002709

PMID:37125655

Abstract

Mitapivat is an orally bioavailable small molecule allosteric activator of pyruvate kinase. It was approved by the US FDA on February 17, 2022, and the European Union in November 2022 for the treatment of hemolytic anemia in adult patients with pyruvate kinase deficiency. In this short perspective, physicochemical properties, synthesis, dosage and administration, mechanism of action, pharmacodynamics, pharmacokinetics, drug interactions, and adverse reactions of mitapivat are described.

摘要

米他匹韦是一种口服生物可利用的丙酮酸激酶小分子变构激活剂。它于2022年2月17日获得美国食品药品监督管理局（FDA）批准，并于2022年11月获得欧盟批准，用于治疗丙酮酸激酶缺乏症成年患者的溶血性贫血。在这篇简短的综述中，描述了米他匹韦的物理化学性质、合成方法、剂量与用法、作用机制、药效学、药代动力学、药物相互作用及不良反应。