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Eculizumab: A Review in Neuromyelitis Optica Spectrum Disorder

Eculizumab: A Review in Neuromyelitis Optica Spectrum Disorder

Journal: Drugs; April 7, 2020

Author(s): James E. Frampton

Review of eculizumab treatment for NMOSD

Eculizumab is the first therapy to have been approved in the EU, USA, Canada, and Japan for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who test positive for aquaporin-4 antibodies and (in the EU only) for those with a relapsing form of disease. This review provides a summary of all the clinical trial findings (the PREVENT study) for eculizumab over 4 years of treatment. Compared to placebo, eculizumab significantly reduced the risk of relapses in NMOSD patients testing positive for AQP4 antibodies; approximately a quarter of these patients were not on any other immunosuppressants while receiving eculizumab. Eculizumab treatments showed improvements in disability and health-related quality-of-life scores across all patient subgroups analyzed. Eculizumab treatment showed a similar long-term safety profile it is known to have for other conditions where it is approved. Thus, eculizumab provides an effective, generally well tolerated and approved treatment option for NMOSD.

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