Ultragenyx announced the submission of a Biologics License Application (BLA) for burosumab as a potential treatment for X-Linked Hypophosphatemia (XLH). Burosumab has previously received Breakthrough Therapy Designation from the Food and Drug Administration (FDA) for the treatment of XLH in pediatric patients ≥1 year of age.
Burosumab is an investigational recombinant fully human monoclonal IgG1 antibody against the phosphorus hormone fibroblast growth factor 23 (FGF23). XLH is rare genetic disorder, characterized by excess levels of FGF23 that reduce serum levels of phosphorus and active vitamin D; burosumab is designed to inhibit FGF23 levels.
A clinical study of burosumab is ongoing, which as well as XLH, is also investigating burosumab as a possible treatment for tumor-induced osteomalacia (TIO).
Burosumab is being developed in a collaboration between Ultragenyx and Kyowa Hakko Kirin Pharmaceuticals, based on a license agreement between both companies.
The FDA will make a decision on the BLA and set a Prescription Drug User Fee Act (PDUFA) action date within the next 60 days.
For more information visit Ultragenyx.com.