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Burosumab in paediatric practice: transforming treatment for X-linked hypophosphataemia
- Dr Sanjay GuptaConsultant paediatrician at Hull University Teaching Hospitals NHS Trust
Key points
- Burosumab is a fully human monoclonal antibody given as a fortnightly subcutaneous injection for X-linked hypophosphataemic rickets.
- Conventional oral phosphate and activated vitamin D have problems with compliance, palatability, gastrointestinal side effects and daily dosing.
- In the speaker's cohort, tolerability has been great, phosphate levels have normalised, bone health is excellent and bone pain has almost disappeared.
Dr Sanjay Gupta discusses the novel treatment for X-linked hypophosphataemia (XLH), focusing on burosumab—a fully human monoclonal antibody that targets the underlying pathology of the disease. Traditionally, XLH has been managed with high doses of oral phosphate and activated vitamin D, treatments that are often challenging due to poor compliance, unpleasant taste, gastrointestinal side effects, and the need for daily administration. Burosumab, administered as a fortnightly subcutaneous injection, addresses the root cause by inhibiting excess fibroblast growth factor 23 (FGF-23), which leads to phosphate loss in urine and reduced activated vitamin D levels. Dr Gupta shares his clinical experience, noting excellent tolerability in children treated with burosumab, normalisation of phosphate levels, improved bone health without the need for further corrective surgery, and significant reduction in bone pain—resulting in a marked improvement in quality of life. This interview took place at the RCPCH conference 2025 in Glasgow, Scotland.
Read the transcript
Automatic transcript, lightly edited for readability. It may contain errors, so check it against the video before quoting.
So of course, when we talked about X-linked hypophosphataemic rickets, it's important to consider the new treatment, which is burosumab. So it's a fully human monoclonal antibody which targets the pathology. So conventionally we treat this condition with high doses of oral phosphate and activated vitamin D. The problem is with the compliance and the palatability and the side effects of phosphate, which is gastrointestinal side effects. And of course it's a daily treatment, whereas burosumab is a subcutaneous injection which is given fortnightly.
The main pathology in X-linked hypophosphataemic rickets is high levels of FGF-23, which is fibroblast growth factor, which leads to phosphate loss in the urine and reduced levels of activated vitamin D. And from my experience within our cohort of children, the tolerability has been great. They haven't needed any more corrective surgery after this, and the bone health is like pristine. And for the first time the phosphate levels have normalised in these children, and a big impact on quality of life: the bone pain which they were experiencing has almost disappeared.
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Dr Sanjay Gupta. “Burosumab in paediatric practice: transforming treatment for X-linked hypophosphataemia.” The Paediatric Video Journal, 4 July 2025. https://paediatricvj.com/videos/burosumab-in-paediatric-practice-transforming-treatment-for-x-gupta/
This interview is educational content intended for healthcare professionals and is not medical advice. Views expressed are those of the speaker. © The Paediatric Video Journal. All rights reserved.