Posted by Michael Wonder on 05 Sep 2018
NICE u-turn approves funding for rare disease therapy Crysvita
5 September 2018 - NICE has changed its position on NHS funding for Kyowa Kirin International’s Crysvita, now endorsing the drug’s use to treat X-linked hypophosphataemia in children and young people with growing bones in England and Wales.
The firm says the positive recommendation “marks a step change in the management of X-linked hypophosphataemia (XLH) for children and young people, and the first significant clinical advancement for the condition in the last 35 years”.
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.
