Alprolix (recombinant Factor IX Fc fusion protein): extended half-life product for the prophylaxis and treatment of hemophilia B

被引:17
作者
Ducore, Jonathan M. [1 ]
Miguelino, Maricel G. [1 ]
Powell, Jerry S. [1 ]
机构
[1] Univ Calif Davis, Med Ctr, Div Hematol & Oncol, Sacramento, CA 95817 USA
关键词
Factor IX; gene therapy; genetic coagulation defects; hemophilia arthropathy; hemophilia B; long-acting factors; prophylaxis; FACTOR PATHWAY INHIBITOR; COAGULATION-FACTOR VIIIA; PREVIOUSLY TREATED PATIENTS; PEGYLATED FACTOR-VIII; HUMAN DOSE TRIAL; PHARMACOKINETIC PROPERTIES; IN-VIVO; PROLONGED ACTIVITY; SAFETY; ALBUMIN;
D O I
10.1586/17474086.2014.951322
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Hemophilia B is a genetic disease caused by mutation of the gene for coagulation protein Factor IX. When severe, the disease leads to spontaneous life-threatening bleeding episodes. Current therapy requires frequent intravenous infusions of therapeutic recombinant or plasma-derived protein concentrates containing Factor IX. Alprolix (TM) (recombinant Factor IX Fc fusion protein), is a therapeutic Factor IX preparation that has been engineered for a prolonged half-life in circulation, has completed pivotal clinical trials and has been approved recently in the USA, Canada, Australia and Japan for use in the clinic for patients with hemophilia B. This promising therapy should allow patients to use fewer infusions to maintain appropriate Factor IX activity levels in all clinical settings, and its use may be indicated in both on demand and prophylactic treatments.
引用
收藏
页码:559 / 571
页数:13
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