The UK’s National Health Service (NHS) is rolling out 鈥檚 Crysvita (burosumab) for the treatment of adults with a rare bone disease called X-linked hypophosphataemia (XLH).
The drug has been available for children with XLH since 2018 but will now be available for eligible adults on the NHS. The service will initially roll the drug out through around 20 specialist centres across England. Additionally, patients will be taught to give Crysvita in their own homes to reduce 鈥渦nnecessary visits to hospitals鈥�.
XLH is a genetic disorder that leads to in the blood. Phosphate is essential for the normal formation of bones and teeth in childhood and helps maintain bone strength in adults. Deficiency of the mineral can result in rickets, an increased risk of fractures, muscle weakness, and dental problems such as abscesses and tooth loss. Patients often experience chronic pain and mobility issues in the most severe cases.聽聽
This move follows the UK鈥檚 National Institute for Health and Care Excellence (NICE) recommendation in August, where the pricing watchdog said that the treatment is a clinically and cost-effective use of NHS resources.
This wasn鈥檛 always the case, as NICE previously rejected the drug for adults on the NHS in draft guidance released in December 2023. In the guidance, NICE commented on pivotal clinical trial data, saying that patients on 鈥渂urosumab may have less pain and fatigue, and improved physical functioning” but added it was 鈥渘ot certain鈥�.
This deliberation came from the results of the Phase III CL303 trial (NCT02526160), which compared Crysvita to a placebo for 24 weeks. After this period, people on placebo switched to Crysvita, where treatment was continued for another 72 weeks. The trial met its primary outcome 鈥� with 94.1% of the treatment arm meeting a mean serum phosphate concentration above the lower limit of normal (2.5mg/dL), compared to 7.6% in the placebo arm. 聽
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By GlobalDataIn the announcement accompanying the roll-out, Oliver Gardiner, trustee at charity XLH UK, said: 鈥淭his is fantastic news for adults with XLH in the UK. This life-changing treatment is the first to tackle the underlying problem and will lessen the significant physical and emotional challenges that adults with XLH face on a daily basis.鈥�
The standard of care for patients with XLH was phosphate supplements or vitamin D, which doesn鈥檛 target the cause of the disease and can cause side effects such as stomach problems and kidney stones. Crysvita works by suppressing the hormone that causes low levels of phosphate and stabilising levels in the blood. According to GlobalData鈥檚 Pharma Intelligence Center, Crysvita will generate $2.4bn in revenue in 2030.
GlobalData is the parent company of Pharmaceutical Technology.
