Children and adolescents with XLH will now be able to access burosumab on the NHS in Scotland
London, England, February 10, 2020. XLH UK, a nonprofit trust representing patients with XLH in the United Kingdom and Ireland, is looking forward to a brighter future now that the Scottish Medicines Consortium (SMC) has enabled access to burosumab (marketed as Crysvita) for X-linked hypophosphataemia (XLH) in children aged 1 and over and in young people with growing bones in Scotland.
XLH is a rare genetic condition inherited as an X-linked dominant trait that causes significant skeletal deformities in children from a young age, with lifelong disability, pain, and fatigue. It is a debilitating condition which can require patients to have multiple, corrective surgeries across the course of their childhood and lifetime.
Burosumab has been recommended by NICE for use across NHS England and Wales since September 2018 and this news from the SMC helps to set a standard of care across the countries. The news is truly life-changing for patients with XLH as it is the first and only treatment that targets the underlying mechanism of their hypophosphataemia. The breakthrough therapy shows improvements in the areas that matter to patients, including reductions in daily pain and stiffness as well as improvements in healing of fractures and rickets severity which may limit the need for invasive, corrective surgeries.
Commenting on SMC’s decision, XLH UK founder and Trustee Oliver Gardiner explained that “This is fantastic news for children with XLH in Scotland. Current therapy is sub-optimal at best and has to be administered multiple times daily. Access to this new life-changing treatment is a first in tackling the underlying problem and has the potential to avoid or mitigate significant physical and emotional challenges.”
We commend Kyowa Kirin International, SMC’s Ultra-Orphan pathway Committee and NHS Scotland for their efforts, dedication and commitment to understand the impact that a rare, hereditary, and lifelong disorder has on individuals and their families. Most importantly we would like to thank all the patients who shared their experiences with us through our 2019 patient survey which helped us immensely in preparing our patient organisation submission for the SMC so that decision-makers could understand what this treatment will mean to patients and their families.