A phase 2 trial of burosumab for treatment of fibroblast growth factor-23 mediated hypophosphatemia in children and adults with fibrous dysplasia.

ABSTRACT: Fibrous dysplasia (FD) is a rare disorder associated with fractures and deformities. FD lesions produce excess phosphaturic hormone fibroblast growth factor 23 (FGF23), leading to hyperphosphaturia in most patients, and hypophosphatemia in those with high FD burden. Skeletal complications are associated with both low-normophosphatemia and frank hypophosphatemia. Burosumab is approved for other forms of FGF23 excess, but there is little evidence to inform use in FD. A phase 2 study investigated the safety and efficacy of burosumab in patients with FD. The primary endpoint was the proportion of participants achieving phosphate levels within a high-normal target range (age and sex-adjusted Z-score -1 to +2). 12 participants (7 children, 5 adults) received burosumab for 48 weeks. Median phosph

SUBMITTER: de Jong O 

PROVIDER: S-EPMC12754703 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature