Burosumab (5) – Crysvita®

• Clinical trials
  • To assess the additional benefit of buurosumab for adults with X-linked hypophosphataemia, the pharmaceutical manufacturer submitted the pivotal, multicentre, randomised, double-blind, placebo-controlled Phase III-trial UX023-CL303, which formed the basis for marketing authorisation, with data cut-off at week 24.

Adults with X-linked hypophosphataemia (XLH)

• The additional benefit is not proven.
• Overall, the pharmaceutical manufacturer did not submit any study for the indicated therapeutic indication that would have been suitable for assessing the additional benefit of burosumab compared with the appropriate comparator therapy.
• An additional benefit of Burosumab compared with the appropriate comparator therapy is therefore not proven.
• The UX023-CL303 study cannot be used for the present benefit assessment, as the appropriate comparator therapy was not implemented.
• The G-BA has defined phosphate replacement and active vitamin D (calcitriol or alfacalcidol) in combination as the appropriate comparator therapy.
• However, in the UX023-CL303 study, phosphate and active vitamin D replacement were explicitly excluded.
• The limited use of phosphate and active vitamin D does not reflect the everyday healthcare situation.
• No contraindications or clinical reasons for omitting phosphate replacement and active vitamin D were provided for the participants in the UX023-CL303 study, meaning that the UX023-CL303 study cannot be used for benefit assessment due to the failure to implement the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.