Emerging treatments
Eculizumab
Eculizumab is a monoclonal antibody that inhibits activation of the terminal component of complement, which is key in the pathogenesis of neuromyelitis optica spectrum disorder (NMOSD). In a randomised, double-blind, phase 3 clinical trial among patients with aquaporin-4 (AQP4)-IgG-positive NMOSD, patients treated with eculizumab had a significantly lower risk of relapse than those who received placebo. There was no significant between-group difference in measures of disability progression.[86] The US Food and Drug Administration (FDA) and the European Medicines Agency have approved eculizumab for treating NMOSD in adult patients who are anti-AQP4 antibody positive. Because of concerns about potentially life-threatening meningococcal infection, eculizumab is available only through a restricted programme under a Risk Evaluation and Mitigation Strategy in the US. Healthcare professionals must counsel patients about the risk and provide educational materials, ensure patients are vaccinated with meningococcal vaccine(s), and monitor patients closely.
Inebilizumab
Inebilizumab, a monoclonal antibody that binds to the B-cell surface antigen CD19, is approved by the FDA for the treatment of NMOSD in adult patients who are anti-AQP4 antibody positive. In a randomised, placebo-controlled, phase 2/3 clinical trial among patients with NMOSD, 21 (12%) of 174 participants receiving inebilizumab had an NMOSD attack, versus 22 (39%) of 56 participants receiving placebo. The rate of occurrence of adverse events was similar in the two groups.[87]
Satralizumab
Satralizumab, a humanised monoclonal antibody targeting the interleukin-6 receptor, is approved by the FDA for the treatment of NMOSD in adult patients who are anti-AQP4 antibody positive. In a randomised, double-blind, phase 3 trial among patients with NMOSD receiving immunosuppressant treatment, relapse occurred in 8 patients (20%) in the satralizumab arm versus 18 (43%) receiving placebo.[88] There was no significant difference in effect between the trial groups in pain and fatigue. In a subsequent phase 3, double-blind, parallel-group trial, relapse occurred in 19 (30%) patients with NMOSD receiving satralizumab monotherapy versus 16 (50%) receiving placebo.[89] In both trials, the rate of serious adverse events and infections were similar between groups.[88][89]
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