SCOTTISH MEDICINES CONSORTIUM (SMC) ENABLES ACCESS TO THE FIRST NEW TREATMENT IN 40 YEARS FOR RARE INHERITED BONE DISORDER IN CHILDREN
Kyowa Kirin International today announced that the SMC has enabled access to Crysvita® (burosumab) in the NHS in Scotland to treat X-linked hypophosphataemia (XLH), in children with radiographic evidence of bone disease, one year of age and older and adolescents with growing skeletons.1
Crysvita is the first treatment to target the underlying mechanism of XLH and represents the first significant clinical advance in nearly 40 years. The SMC’s assessment means that Crysvita is one of the first medicines to be evaluated under the ultra-orphan pathway, a new system for the consideration of medicines for extremely rare diseases. Under this novel framework, eligible Scottish children will be able to access Crysvita, whilst Kyowa Kirin collects additional relevant data to address areas of uncertainty within this rare disease. The SMC will review this further evidence after three years and make a final decision on its routine use in NHS Scotland.2
XLH is an inherited genetic disorder that causes low levels of phosphate in the blood. This leads to bone and skeletal issues that can result in inadequate growth, life-long physical disabilities and pain. Children with the condition usually have bowed or bent legs, short stature, bone pain and delayed walking, fatigue, 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 (EMA) in February 2018.3
Oliver Gardiner, Founder and Trustee of XLH UK, said: “This is important news for children and young adults with XLH in Scotland, who will now be able to benefit from Crysvita. 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 people with XLH and their families.”
The SMC’s initial assessment is based on data including the pivotal Phase 3 UX023-CL301 study in which 61 children (aged 1-12 years) with XLH were randomised to be treated with either Crysvita or conventional therapy with oral phosphate and active Vitamin D. A significant improvement in the primary endpoint of rickets healing at 40 weeks was shown versus conventional treatment, and improvements were observed in serum phosphate levels, linear growth, and physical function.4
“Kyowa Kirin International is committed to improving the lives of the children across Europe who are living with XLH”, said Abdul Mullick, President, Kyowa Kirin International. “Access to Crysvita marks a step change in the management of children and adolescents in Scotland with XLH, and we look forward to collecting more data on the efficacy and tolerability of the treatment over the next few years.”