NICE recommends Crysvita for treatment of rare bone disorder in young patients

The National Institute for Health and Care Excellence (NICE) has given a positive recommendation for Crysvita (burosumab) to be used in the treatment of X-linked hypophosphataemia (XLH) in children and young people with growing bones.

XLH is a rare, inherited, genetic bone disorder that causes low levels of phosphates in the blood, leading to soft, weak bones. It can lead to life-long physical disabilities and pain.

Kyowa Kirin International’s Crysvita is an anti-FGF23 fully human monoclonal antibody and the first treatment to target the underlying pathophysiology of XLH. It was granted a conditional marketing authorisation from the European Medicines Agency (EMA) in February this year (2018).

“This is important news for children and young adults with XLH who will now be able to benefit from Crysvita routinely on the NHS,” said Oliver Gardiner, board member at XLH UK. “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.”

“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,” added Dr Poonam Dharmaraj, chairperson of the British Paediatric and Adolescent Bone Group. “It will result in better healing of rickets, linear growth and muscle function among affected individuals.”

“Kyowa Kirin International is committed to improving the lives of the many children across Europe who are living with XLH,” stressed Tom Stratford, CEO, Kyowa Kirin International. “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.”