| Description |
Asfotase alfa (ENB-0040) is a bone-targeted genetically engineered glycoprotein. Asfotase alfa increases the survival rate, bone mineralization and bone length and prevents mineralization defects of the feet, rib cage, lower limbs, jaw bones in Akp2−/− knockout mice. Asfotase alfa can be used for the research of perinatal, infantile, and juvenile-onset hypophosphatasia (HPP)[1].
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| In Vivo |
Asfotase alfa (0.5-8.2 mg/kg; s.c. once daily for 43 days) shows dose-response relationships that strongly support the pharmacological efficacy for HPP[1]. Animal Model: Akp2−/− knockout mice[1] Dosage: 0.5, 2.0 and 8.2 mg/kg Administration: Subcutaneous injection; once daily for 43 days Result: Dose-dependently increased the survival rate of mice, bone mineralization and prevention of mineralization defects of the feet, rib cage, lower limbs, jaw bones, the median survival, body weight, and bone length. Showed a clear relationship between daily dose and achieving a healthy BW and the ED80 (the dose that prevents bone defects in 80% of mice) was 3.2, 2.8 and 2.9 mg/kg/day for these sites. Remained increasing urinary PPi concentrations in all treatment groups.
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