NICE final guidance recommends Crysvita for XLH

The National Institute for Health and Care Excellence (NICE) has issued final guidance recommending Crysvita (burosumab) within its marketing authorization, for treating X-linked hypophosphatemia (XLH) in children and young people with growing bones in England and Wales, from the Japanese drugmaker Kyowa Kirin (TYO: 4151).

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 authorization from the European Medicines Agency in February 2018. The US Food and Drug Administration approved the drug in April this year.