Scottish Medicines Consortium enables access to treatment for rare inherited bone disorder in children
pharmafile | February 18, 2020 | News story | Manufacturing and Production | Crysvita, Kyowa Kirin, SMC, Scottish Medicine Consortium, nhs scotland
The Scottish Medicines Consortium (SMC) has enabled access to Kyowa Kirin’s Crysvita (burosumab) for the NHS in Scotland to treat X-linked hypophosphataemia (XLH), in children with radiographic evidence of bone disease.
Crysvita is an anti-FGF23 fully human monoclonal antibody, and the first treatment to target the underlying pathophysiology of XLH.
XLH is an inherited genetic disorder that causes low levels of phosphate in the blood, which leads to bone and skeletal issues. This can result in inadequate growth and life-long disabilities. In children this usually causes bone pain, hearing loss, short stature, bowed or bent legs, fatigue, dental problems and hearing loss.
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 a pivotal Phase 3 study in which 61 children, aged between 1-12 years, with XLH were randomized to be treated with either Crysvita or conventional therapy with oral phosphate and active Vitamin D.
Abdul Mullick, President of Kyowa Kirin International, said: “Kyowa Kirin International is committed to improving the lives of the children across Europe who are living with XLH. 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.”
Conor Kavanagh
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