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NICE recommends first new X-linked hypophosphataemia therapy in over 30 years

Published on 10/10/18 at 11:13am

NICE has announced its evidence-based recommendation that Kyowa Kirin International’s Crysvita (burosumab) be made available on the NHS for the treatment of X-linked hypophosphataemia (XLH) in children and young people with growing bones in England and Wales, giving patients the first new advancement for the condition for 35 years.

An inherited genetic disorder, XLH is characterised by the ineffective reabsorption of phosphate into the bloodstream, leading to weakened and softened bones and ultimately life-long pain and disability. This can include bowed or bent legs, delayed walking, bone pain, dental problems and hearing loss.

Kyowa Kirin International says that its anti-FGF23 fully human monoclonal antibody is “the first treatment to target the underlying pathophysiology” of the condition. Thus far it has scored a conditional marketing authorisation from the European Medicines Agency back in February. Following NICE’s recommendation, the manufacturer will extend its existing Early Access Programme in the UK to allow time for the final guidance to be implemented.

“This is important news for children and young adults with XLH who will now be able to benefit from Crysvita routinely on the NHS,” commented 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.”

Dr Poonam Dharmaraj, Chairperson of the British Paediatric and Adolescent Bone Group said the decision would “result in better healing of rickets, linear growth and muscle function among affected individuals,” while Tom Stratford, CEO of Kyowa Kirin International, remarked: “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.”

Matt Fellows

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