A review of the application of asfotase alfa in the rare disease hypophosphatasia
LIU Qing-yang
HOU Zi-jing
GE Yu
WANG Shao-hong
LIU Xin
ZHANG Bo
TANG Yan
Abstract：Hypophosphatasia(HPP)is a rare hereditary endocrine system disease characterized by bone and/or tooth mineralization disorders accompanied by decreased serum alkaline phosphatase activity.Asfotase alfa(AA)received orphan drug approval by FDA as a recombinant bone-targeted human nonspecific alkaline phosphatase and was marketed in 2015 for the treatment of HPP.This paper reviews the mechanism of action,pharmacokinetic characteristics,efficacy,safety,usage and dosage of AA to provide reference and evidence support for the use of AA in the patients with HPP.
Keywords：hypophosphatasiarare diseaseorphan drugasfotase alfa
Publication Date：2023-12-31
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：5( 2526-2530 )