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The National Institute for Health and Care Excellence (NICE) recently recommended making burosumab available for all adults with X-linked hypophosphataemia (XLH), a rare inherited disorder characterised by low levels of phosphate in the blood leading to bone and tooth problems.
Clinical trial evidence shows that burosumab effectively increases the level of phosphate in the blood resulting in less pain and fatigue, and physical improvements, but this was restricted to adults aged from 18 to 65 years with at least 4 out of 10 pain. Critically, evidence for teenagers between stopping growth and becoming 18 years of age is lacking.
With funding from Kyowa Kirin International, researchers at NDORMS will look at the longer term use of burosumab and whether it improves the lives of all adults living with the condition as well as measure the impact of XLH on the lives of caregivers.