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Hypophosphatasia Medication

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

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]


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.

Contributor Information and Disclosures

Horacio B Plotkin, MD, FAAP Chief Medical Officer, Retrophin, Inc; Adjunct Associate Professor of Pediatrics and Orthopedic Surgery, University of Nebraska College of Medicine

Horacio B Plotkin, MD, FAAP is a member of the following medical societies: American Academy of Pediatrics

Disclosure: Received salary from Retrophin, Inc for management position.


George A Anadiotis, DO Consulting Staff, Department of Pediatric Rehabilitation and Development, Division of Clinical and Biochemical Genetics, Emmanuel Children's Hospital

George A Anadiotis, DO is a member of the following medical societies: American Medical Association, American Society of Human Genetics

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

Chief Editor

Luis O Rohena, MD Chief, Medical Genetics, San Antonio Military Medical Center; Assistant Professor of Pediatrics, Uniformed Services University of the Health Sciences, F Edward Hebert School of Medicine; Assistant Professor of Pediatrics, University of Texas Health Science Center at San Antonio

Luis O Rohena, MD is a member of the following medical societies: American Academy of Pediatrics, American Chemical Society, American College of Medical Genetics and Genomics, American Society of Human Genetics

Disclosure: Nothing to disclose.

Additional Contributors

James Bowman, MD Senior Scholar of Maclean Center for Clinical Medical Ethics, Professor Emeritus, Department of Pathology, University of Chicago

James Bowman, MD is a member of the following medical societies: Alpha Omega Alpha, American Society for Clinical Pathology, American Society of Human Genetics, Central Society for Clinical and Translational Research, College of American Pathologists

Disclosure: Nothing to disclose.

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