Hypophosphatasia Medication

Updated: Dec 11, 2015
  • Author: Horacio B Plotkin, MD, FAAP; Chief Editor: Luis O Rohena, MD, FAAP, FACMG  more...
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Medication

Medication Summary

Until 2015, no effective drug therapy had been approved for hypophosphatasia. The FDA approved asfotase alfa as the first-ever therapy for hypophosphatasia caused by a rare hereditary mutation in alkaline phosphatase gene. [7, 8]

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Enzymes, Metabolic

Class Summary

Enzyme replacement therapy helps to prevent bone demineralization.

Asfotase alfa (Strensiq)

Enzyme replacement that is a soluble glycoprotein composed of 2 identical polypeptide chains; each chain consists of the catalytic domain of human tissue–nonspecific alkaline phosphatase (TNSALP), the human immunoglobulin G1 Fc domain and a deca-aspartate peptide used as a bone-targeting domain. It is indicated for perinatal/infantile- and juvenile-onset hypophosphatasia.

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