UK – XLH patients to get routine access to first new therapy in 30 years

For the first time in more than 30 years children and young adults in England and Wales with the rare brain disorder XLH will have access to a new treatment option via the NHS.

The National Institute for Health and Care Excellence has published final guidance recommending use of Kyowa Kirin’s Crysvita (burosumab) within its marketing authorisation, that is for treating X-linked hypophosphataemia (XLH) in children and young people with growing bones.

There are around 250 children and young people in England with XLH, an inherited genetic disorder that causes low levels of phosphate in the blood. This leads to soft, weak bones, and 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.

Current treatment mainly consists of vitamin D supplementation and oral phosphate, and is aimed at improving growth, lessening the severity of symptoms and preventing skeletal abnormalities…