Emerging Role of Targeted Monoclonal Antibodies in Neuromyelitis Optica Spectrum Disorders

Neuromyelitis optica spectrum disorder (NMOSD) is a rare, autoimmune disease that results in recurring, often severe attacks on the optic nerves and spinal cord that may result in profound visual loss and paralysis. Since the late 1990s, NMOSD was treated with traditional immunosuppressive therapies. Recently, four monoclonal antibodies (mAbs) were granted Food and Drug Administration approval for aquaporin-4 antibody (AQP4-IgG)-seropositive NMOSD treatment: eculizumab, inebilizumab, satralizumab, and ravulizumab. These targeted immunomodulatory therapies have emerged as a transformative approach to effectively reduce NMOSD attack frequency in AQP4-IgG-seropositive patients. We explore the role of these preventative mAbs for NMOSD management by reviewing the efficacy, safety, mechanisms of action, and administration of these agents, and compare them to rituximab and traditional immunosuppressants. We discuss therapy selection and the clinical challenges of therapeutic management, including medication adherence, therapeutic monitoring strategies, economic considerations, and medication accessibility, while managing therapy failure and indications for transitioning to alternative therapies.