Ravulizumab (5) – Ultomiris®

• Clinical trials
  • The ALXN1210-NMO-307 trial is an ongoing, single-arm, externally placebo-controlled, open-label trial of ravulizumab in adults with NMOSD, who are AQP4 antibody-seropositive and have experienced at least one relapse in the 12 months prior to study enrolment.
  • The ECU-NMO-301 trial is a completed, double-blind, randomised, placebo-controlled trial of eculizumab in adults with NMOSD, who are AQP4 antibody-seropositive and who have experienced at least 2 relapses in the 12 months prior to study enrolment, or at least 3 relapses within the 24 months prior to study enrolment, with at least 1 relapse in the 12 months prior to study enrolment.

Adults with neuromyelitis optica spectrum disorders (NMOSD) who are anti-aquaporin-4-IgG (AQP4-IgG) seropositive

• For adults with neuromyelitis optica spectrum disorders (NMOSD) who are anti-aquaporin-4-IgG (AQP4-IgG) seropositive, the additional benefit is not proven.
• Overall, therefore, there are no data available suitable for the benefit assessment of ravulizumab, meaning that additional benefit is not proven.
• In the absence of direct comparative studies against an active ingredient (INN) used in the appropriate comparator therapy, the pharmaceutical manufacturer has submitted two indirect comparisons for the benefit assessment: a comparison of individual arms from different studies of ravulizumab and eculizumab using a propensity score procedure, and a comparison of ravulizumab with both eculizumab and satralizumab via a network meta-analysis.
• In the comparison using the propensity score procedure, the methodology and approach adopted by the pharmaceutical manufacturer are inadequate. The data submitted are therefore not interpretable.
• Nor are the analyses presented from the network meta-analysis suitable for assessing the additional benefit.
• Overall, there are therefore no suitable data available for Ravulizumab compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.