Drugs & Therapy Perspectives

, Volume 34, Issue 11, pp 497–506 | Cite as

Burosumab in X-linked hypophosphatemia: a profile of its use in the USA

  • Katherine A. Lyseng-Williamson
Adis Drug Q&A


Burosumab (Crysvita®), a fully human IgG1 monoclonal antibody directed at fibroblast growth factor 23 (FGF23), is indicated for the treatment of X-linked hypophosphatemia (XLH), a condition associated with excessive FGF23 production. It directly addresses the excessive FGF23 activity in patients with XLH by binding to FGF23, and inhibiting its signaling. This leads to increased gastrointestinal phosphate absorption and renal phosphate reabsorption, thereby improving serum phosphate levels, and, ultimately, bone mineralization and the risk of bone disease. In clinical trials, subcutaneous burosumab increased serum phosphorus levels in pediatric and adult patients with XLH, as well as significantly improving the severity of rickets in children, and improving pain, stiffness, physical functioning, and fracture/pseudofracture healing in adults. Burosumab is well tolerated by children and adults with XLH, with most treatment-emergent adverse events being of mild to moderate severity.



The manuscript was reviewed by: S. Fukumoto, Department of Molecular Endocrinology, Fujii Memorial Institute of Medical Sciences, Tokushima University, Tokushima, Japan; L.-N. Veilleux, Motion Analysis Center, Shriners Hospital for Children-Canada, Montréal, QC, Canada. During the peer review process, Ultragenyx Pharmaceutical Inc., the marketing-authorization holder of burosumab, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.

Compliance with ethical standards


The preparation of this review was not supported by any external funding.

Conflict of interest

K.A. Lyseng-Williamson is an employee of Adis/Springer, is responsible for the article content and declares no conflicts of interest.


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© Springer Nature Switzerland AG 2018

Authors and Affiliations

  1. 1.SpringerAucklandNew Zealand

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