Burosumab (also known as the drug, Crysvita®) is a fully human immunoglobulin G1 (IgG1)
monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor
23 (FGF23), leading to an increase in serum phosphorus levels. This drug is already approved
for use in patients with X-linked hypophosphatemia (XLH), but not for Cutaneous Skeletal
Hypophosphatemia Syndrome (CSHS). It is hypothesized that burosumab may provide clinical
benefit to a patient with CSHS due to the common underlying feature in this patient and in
patients with XLH - abnormally elevated FGF23 in the context of low age -adjusted serum
phosphorous levels.
Phase:
Early Phase 1
Details
Lead Sponsor:
Laura Tosi
Collaborators:
Children's National Research Institute Children's Research Institute Ultragenyx Pharmaceutical Inc