Summary

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• Ravulizumab-cwvz (Ultomiris) is recommended for adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody positive, and neuromyelitis optica spectrum disorder in adults who are anti-aquaporin-4 antibody positive.
• The analysis on Ultomiris involved six systematic reviews/meta-analyses, comparing Ultomiris with other treatments focusing on safety, efficacy, and applicability among different population types, including subgroups with the indicated conditions such as NMOSD, aHUS, PNH, and gMG.
• In the case of NMOSD patients seropositive for anti-AQP4 antibodies, Ultomiris significantly reduced the rate of the first adjudicated relapse while enhancing disability levels and quality of life. Its safety profile was comparable to placebo, indicating good tolerability.
• In the treatment of aHUS, Ultomiris and Eculizumab were equally effective. Both treatments play crucial roles in inhibiting the complement cascade integral to the pathogenesis of aHUS, with similar safety profiles. However, there was a preference for Ultomiris due to its lower financial impact and less frequent dosing requirements.
• For managing PNH compared to Eculizumab or the C3 inhibitor pegcetacoplan, Ultomiris showed substantial efficacy by reducing hemolysis, as measured by LDH levels, increasing hemoglobin levels, and preventing the need for transfusions, thereby further improving the quality of life and demonstrating comparable safety over various treatment durations.
• Ultomiris did not show a significant difference in effectiveness against gMG when compared with FcRn treatments like efgartigimod and rozanolixizumab in improving Myasthenia Gravis-Activities of Daily Living scale (MG-ADL) scores. However, FcRn treatments had a greater short-term effect on the Quantitative Myasthenia Gravis scale (QMG) scores.