Does FGF23 Inhibition Improve Outcomes in X-Linked Hypophosphatemic Rickets?

Burosumab, a human monoclonal antibody against FGF23, was more effective than standard phosphate and vitamin D therapy in children with X-linked hypophosphatemia.

X-linked hypophosphatemia is the most common genetic cause of rickets and is associated with profound renal phosphate wasting, growth failure, and elevated levels of fibroblast growth factor 23 (FGF23).

To test whether burosumab, a fully human monoclonal antibody against FGF23, would be more effective than standard phosphate and vitamin D therapy in treating and healing rickets, investigators conducted an industry-funded, international, open-label, randomized, active-controlled phase III trial involving 61 children (aged, 1–12 years) with X-linked hypophosphatemia who were receiving standard therapy. Half of the patients continued standard therapy and half were switched to receive burosumab subcutaneous injections every 2 weeks for 64 weeks.…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

Does FGF23 Inhibition Improve Outcomes in X-Linked Hypophosphatemic Rickets?

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Reviewing Author