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Clinical trial assesses anti-FGF23 for treating X-linked hypophosphatemia

() is a heritable form of rickets that results from mutations in the gene encoding the phosphate regulating endopeptidase (PHEX).

Unlike diet-associated forms of rickets, XLH cannot be ameliorated by vitamin D ingestion. XLH patients have increased serum levels of FGF23, which decreases both inorganic phosphate (Pi) and the activated form of vitamin D.

In this issue of the , and colleagues at Yale University evaluated the effectiveness and safety of an anti-FGF23 antibody (KRN23) in a small cohort of XLH patients. A single dose of KRN23 administered intravenously (i.v.) or subcutaneously (s.c.) improved renal phosphate reabsorption, and increased serum Pi and activated vitamin D concentrations, and s.c. administration provided benefit for a longer duration than i.v. dosing.

These findings, along with a favorable safety profile, indicate that KRN23 should be further evaluated for use in XLH patients.

TITLE: Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia

Source

Journal of Clinical Investigation