Rare Daily Staff

The National Institute for Health and Care Excellence, the United Kingdom’s drug value watchdog, has issued a positive recommendation for Kyowa Kirin International’s Crysvita for treating X-linked hypophosphataemia (XLH) in children and young people with growing bones in England and Wales.

The final guidance, expected to be published October 24, represent a reversal of NICE’s initial opinion on Cryvita.

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.

“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,” said Poonam Dharmaraj, chair 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 has been providing access to Crysvita for eligible patients at no cost, via an early access program in the United Kingdom. The program will be extended to allow time for NHS England to implement the NICE final guidance.

September 5, 2018

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