Burosumab Significantly Improves Symptoms in Children With X-Linked Hypophosphatemia
A study including 61 children (aged 1 to 12 years) with X-lined hypophosphatemia showed significantly greater clinical improvements in disease-related symptoms, including rickets severity, growth, and biochemistry panels, among those treated with burosumab (Crysvita®) compared with those continuing conventional therapy with oral phosphate and active vitamin D.1 By week 40, 72% of burosumab-treated children showed substantial healing of their rickets compared with 6% of those treated with conventional therapy. Additionally, burosumab-treated children demonstrated greater improvements in growth, leg deformities, distance walked in 6 minutes, and serum phosphorous and active vitamin D levels.1
Burosumab is a fully human monoclonal antibody against the protein fibroblast growth factor 23 (FGF23), a bone-derived hormone that regulates phosphate metabolism. Based on the positive findings of 4 clinical trials, burosumab was approved by the US Food and Drug Administration in 2018 to treat adults and children aged ≥1 year with x-linked hypophosphatemia, making it is the first therapy approved for this orphan disease.2 Researchers will continue to assess burosumab to determine whether it will impact children’s height outcomes as they reach adulthood and reduce the need for corrective surgery for rickets-related deformities, such as bowed legs.
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1. Imel EA, Glorieux FH, Whyte MP, et al. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial. Lancet. 2019 May 16. pii: S0140-6736(19)30654-3. doi: 10.1016/S0140-6736(19)30654-3.
2. US Food and Drug Administration. FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia. Released April 17, 2018. Accessed June 14, 2018.