Ravulizumab (4) – Ultomiris®

• Clinical trials
  • The CHAMPION trial was a double-blind, randomised controlled trial in which the efficacy and safety of ravulizumab were compared with those of placebo – in each case, where appropriate, in combination with standard treatment – over a period of 26 weeks.
  • The REGAIN study is a randomised, controlled, double-blind, phase 3 study comparing treatment with eculizumab against placebo – in each case, where appropriate, in addition to standard treatment – over 26 weeks.

Adults with acetylcholine receptor antibody-positive generalised myasthenia gravis who are eligible for add-on therapy to standard treatment

• For adults with acetylcholine receptor antibody-positive generalised myasthenia gravis who are eligible for adjunctive therapy to standard treatment, the additional benefit is not proven.
• An additional benefit is therefore not proven.
• Overall assessment
  • For the benefit assessment of ravulizumab for the treatment of AChR-Ab-positive adults with generalised myasthenia gravis as an add-on therapy to standard treatment, the results of the Phase 3 CHAMPION trial, as well as an indirect comparison based on this trial and the REGAIN trial on eculizumab.
  • In the CHAMPION study, the efficacy and safety of ravulizumab were compared with those of placebo – in each case, where applicable, in addition to standard therapy. Consequently, there are no direct comparative data available for ravulizumab against the appropriate comparator therapy adjusted for this procedure.
  • Furthermore, the indirect comparison presented by the pharmaceutical manufacturer, using IPW via the bridge comparator placebo to compare ravulizumab with eculizumab in order to derive the additional benefit for the patient population of treatment-refractory patients, is not suitable, as it cannot be assumed that the study populations are sufficiently similar.
  • For adults with AChR-AK-positive generalised myasthenia gravis who are eligible for add-on therapy to standard treatment, there are therefore no suitable data available to assess the additional benefit of ravulizumab compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.