1.1
Burosumab is recommended, within its marketing authorisation, as an option for treating X‑linked hypophosphataemia (XLH) in adults. Burosumab is only recommended if the company provides it according to the commercial arrangement.
Burosumab is recommended, within its marketing authorisation, as an option for treating X‑linked hypophosphataemia (XLH) in adults. Burosumab is only recommended if the company provides it according to the commercial arrangement.
Why the committee made these recommendations
Usual treatment for XLH in adults is oral phosphate and active vitamin D. Burosumab is used in the NHS for treating XLH in people under 18; this evaluation is for treating XLH in adults.
Clinical trial evidence shows that burosumab increases the level of phosphate in the blood more effectively than placebo. The evidence also suggests that people having burosumab may have less pain and fatigue, and improved physical functioning compared with placebo in the short term, but this is uncertain.
Although there are some uncertainties in the economic model, the cost-effectiveness estimates are within the range considered an acceptable use of NHS resources. So, burosumab is recommended.