The positive recommendation marks a step change in the management of XLH for children and young people, and the first significant clinical advancement for the condition in the last 35 years.
LONDON, UNITED KINGDOM, 5th September 2018: The National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for Kyowa Kirin International’s Crysvita (burosumab) within its marketing authorisation, for treating X-linked hypophosphataemia (XLH) in children and young people with growing bones in England and Wales.1 The final guidance is expected to be published on 24th October 2018.
XLH is an inherited genetic disorder that causes low levels of phosphate in the blood. This leads to soft, weak bones, which can result in life-long physical disabilities and pain. Children with the condition usually have bowed or bent legs, short stature, bone pain and delayed walking, and may also have dental problems and hearing loss.
Crysvita is an anti-FGF23 fully human monoclonal antibody, and the first treatment to target the underlying pathophysiology of XLH. It received a conditional marketing authorisation from the European Medicines Agency in February 2018.2