The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for this medicine using the ultra-orphan framework.
Indication under review: Treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.
- X-linked hypophosphataemia is a chronic, progressive, debilitating multisystem disease. Affected patients have skeletal abnormalities and the main clinical consequence in children is rickets.
- In short-term (64-week) clinical studies in patients aged 1 to 12 years, burosumab demonstrated greater improvement in a radiological measure of rickets, compared with conventional therapy of oral phosphate and vitamin D.
- Whilst a treatment effect on correction of bone defects in childhood has been shown, it is unclear how this will affect progression of bone disease into adulthood and long-term consequences of X-linked hypophosphataemia.
- Some short-term data on quality of life are available but are difficult to interpret.
- An economic evaluation projected significant improvements in quality-adjusted life years over a patient’s lifespan compared with conventional therapy. However, there were uncertainties particularly surrounding quality of life estimates and an assumed ongoing response to burosumab.
- Despite a Patient Access Scheme (PAS), the treatment’s cost in relation to its health benefits remains high.
From February 2020 burosumab can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated. After 3 years the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.
- Medicine name:
- burosumab (Crysvita)
- SMC ID:
For the treatment of X-linked hypophosphataemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.
- Pharmaceutical company
- Kyowa Kirin
- BNF chapter
- Endocrine system
- Submission type
- Ultra-orphan initial assessment
- Date Published
- 10 February 2020