Ultra Orphan Assessment

The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for this medicine using the ultra-orphan framework.

burosumab (Crysvita)

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.

Key points:

  • 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.

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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 details

Medicine name:
burosumab (Crysvita)
SMC ID:
SMC2240
Indication:

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
Additional notes

A new approach for assessing medicines for extremely rare conditions started in October 2018. Further information is available on our ultra-orphan webpage and in the Scottish Government’s guidance.