NHS backs childhood X-linked hypophosphatemia (XLH) drug

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.

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.

Commenting on NICE’s decision, Oliver Gardiner, Board Member at XLH UK, said: “This is important news for children and young adults with XLH who will now be able to benefit from Crysvita routinely on the NHS.  Access to a treatment that tackles the underlying mechanism and has the potential to avoid or mitigate substantial physical and emotional challenges, will truly make a difference to the lives of patients and their families.”

Dr Poonam Dharmaraj, Chairperson of the British Paediatric and Adolescent Bone Group said on behalf of the group: “This treatment represents a significant improvement for a condition in which there have been no advances in management for 35 years and will be much easier to adhere to compared to current therapy options. It will result in better healing of rickets, linear growth and muscle function among affected individuals.”

Tom Stratford, CEO, Kyowa Kirin International said: “Kyowa Kirin International is committed to improving the lives of the many children across Europe who are living with XLH. It is a major development that NICE has recommended Crysvita for routine use among children and young people with XLH in England and Wales. This marks a step change in treatment for XLH, emphasised through the emotional testimonies provided by patient groups and clinicians following the first evaluation consultation.”