Pegunigalsidase alfa-iwxj,a long-acting new drug for the treatment of adult Fabry disease patient
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(t1/2)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.
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