Eculizumab Therapy in Neuromyelitis Optica Spectrum Disorder

Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease that causes recurrent inflammation in the central nervous system which has poor prognosis without appropriate treatment. Complement activation mediated by aquaporin-4 antibodies play a main role in pathogenicity of NMOSD. Recently, four novel drugs targeting specific pathomechanism of NMOSD, eculizumab, satralizumab, inebilizumab, and ravulizumab, were introduced in worldwide and the results of randomized controlled trial of these rising stars were encouraging. Eculizamb, inhibitor of the complement system (C5 protein), was approved for their use in seropositive NMOSD in Republic of Korea since April 2024. Here, we introduce the mechanism of action of eculizumab, and its clinical data to evaluate its therapeutic value.