Mitapivat sulfate(Pyrukynd)

被引:0
|
作者
张冰 [1 ]
王绍杰 [1 ]
机构
[1] 沈阳药科大学
关键词
D O I
10.14142/j.cnki.cn21-1313/r.2022.10.009
中图分类号
R977 [影响生长代谢机能药物];
学科分类号
1007 ;
摘要
2022年2月17日,Agios制药公司研发的丙酮酸激酶激活剂mitapivat sulfate经美国FDA批准上市,商品名为Pyrukynd,用于治疗丙酮酸激酶缺乏的成人溶血性贫血。丙酮酸激酶缺乏症是一种罕见的常染色体隐性遗传性疾病,表现为丙酮酸激酶活性降低,并出现慢性溶血性贫血,伴有无效红细胞生成的迹象。
引用
收藏
页码:818 / 818
页数:1
相关论文
共 3 条
  • [1] Mitapivat, a novel pyruvate kinase activator, for the treatment of hereditary hemolytic anemias[J] . Al Samkari Hanny,van Beers Eduard J..Therapeutic Advances in Hematology . 2021
  • [2] The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model
    Matte, Alessandro
    Federti, Enrica
    Kung, Charles
    Kosinski, Penelope A.
    Narayanaswamy, Rohini
    Russo, Roberta
    Federico, Giorgia
    Carlomagno, Francesca
    Desbats, Maria Andrea
    Salviati, Leonardo
    Leboeuf, Christophe
    Valenti, Maria Teresa
    Turrini, Francesco
    Janin, Anne
    Yu, Shaoxia
    Beneduce, Elisabetta
    Ronseaux, Sebastien
    Iatcenko, Iana
    Dang, Lenny
    Ganz, Tomas
    Jung, Chun-Ling
    Iolascon, Achille
    Brugnara, Carlo
    De Franceschi, Lucia
    [J]. JOURNAL OF CLINICAL INVESTIGATION, 2021, 131 (10):
  • [3] Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
    Grace, Rachael F.
    Rose, Christian
    Layton, D. Mark
    Galacteros, Frederic
    Barcellini, Wilma
    Morton, D. Holmes
    van Beers, Eduard J.
    Yaish, Hassan
    Ravindranath, Yaddanapudi
    Kuo, Kevin H. M.
    Sheth, Sujit
    Kwiatkowski, Janet L.
    Barbier, Ann J.
    Bodie, Susan
    Silver, Bruce
    Hua, Lei
    Kung, Charles
    Hawkins, Peter
    Jouvin, Marie-Helene
    Bowden, Chris
    Glader, Bertil
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2019, 381 (10): : 933 - 944