burosumab (Crysvita)

following a full submission assessed under the ultra-orphan process:

Indication under review: Treatment of X-linked hypophosphataemia in adults.

Key points:

• X-linked hypophosphataemia is a rare genetic condition. In adults, symptoms include pathological fractures or pseudofractures with impaired healing, early osteoarthritis, dental problems and an increased risk of osteoporosis. These symptoms are associated with pain and stiffness, may limit physical function and impact quality of life.
• In a phase III study, burosumab significantly increased the proportion of patients achieving mean serum phosphate above the lower level of normal, compared with placebo over 24 weeks of treatment. There is uncertainty relating to the quality of life data.
• Only open-label and uncontrolled data (for less than 3 years of treatment) are available to support burosumab benefits in relation to phosphate levels and symptoms in the long term with continuous treatment. There is uncertainty about long-term efficacy and safety of burosumab, a potentially lifelong treatment, and there are no data to support a mortality benefit with burosumab.
• The submitting company has positioned burosumab for use in symptomatic adult patients (≥18 years old). There is a lack of data against conventional therapy. Uncertainty remains about the relative effectiveness of burosumab against this relevant comparator.
• The cost of burosumab in relation to its health benefits remains high and there are outstanding uncertainties relating to clinical and quality of life data used in the mode

_________________________________________________________________________________________________________________________

From 16 March 2023 burosumab (Crysvita) can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated. At the end of the data collection period the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.