用于成年患者法布雷病治疗的长效制剂pegunigalsidase alfa-iwxj  

Pegunigalsidase alfa-iwxj,a long-acting new drug for the treatment of adult Fabry disease patient

作　　者：刘彬 杜小莉[1] LIU Bin;DU Xiao-li(Department of Pharmacy,Peking Union Medical College Hospital,Chinese Academy of Medical Science,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医院药剂科,北京100730

出　　处：《中国新药杂志》2024年第18期1875-1878,共4页Chinese Journal of New Drugs

摘　　要：法布雷病(Fabry disease,FD)是一种与X染色体关联的伴性遗传的溶酶体贮积病,收录在我国第一批罕见病目录(2018)中。pegunigalsidase alfa-iwxj(商品名:Elfabrio^(TM))是美国FDA批准的第1款用于成年患者法布雷病治疗的长效制剂,其在体内平均半衰期长达80 h,仅需每2周静脉输注1次。临床研究证实,pegunigalsidase alfa-iwxj对成年法布雷病患者的心肾功能改善效果不劣于第1代酶替代治疗(enzyme replacement therapy,ERT)药物。在使用期间需注意监测药品不良反应的发生以及抗药性抗体的滴度。本文就pegunigalsidase alfa-iwxj的作用机制、药动学、临床疗效、安全性等方面进行述评。Fabry disease(FD)is a lysosomal storage disease related to X-linked gene mutation which is included in the first batch of rare disease catalogues in China(2018).Pegunigalsidase alfa-iwxj(tradename:ElfabrioTM)is a long-acting new drug approved by FDA for the treatment of Fabry disease in adult patients.Its mean plasma half-life(t_(1/2))is around 80 hours,and it only needs to be injected one time every two weeks.According to the data from clinical trials,pegunigalsidase alfa-iwxj shows noninferiority compared to the first-generation enzyme replacement therapy(ERT).Care should be taken to monitor the occurrence of adverse reactions and the titer of resistant antibodies during the treatment.In this article,the mechanism of action,pharmacokinetics,and clinical efficacy and safety of pegunigalsidase alfa-iwxj were reviewed.

关 键 词：法布雷病 溶酶体贮积症 酶替代疗法 长效制剂 pegunigalsidase alfa-iwxj 

分 类 号：R977.3[医药卫生—药品]