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Emerging Targeted Therapies for Neuromyelitis Optica Spectrum Disorders

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Abstract

Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune, inflammatory disorder of the central nervous system that typically presents with recurrent episodes of optic neuritis, longitudinally extensive myelitis, brainstem, diencephalic, and cerebral syndromes. Up to 80% of NMOSD patients have a circulating pathogenic autoantibody that targets the water channel aquaporin-4 (AQP4-IgG). The discovery of AQP4-IgG transformed our understanding of the pathogenesis of the disease and its possible treatment targets. Monoclonal antibodies targeting terminal complement (eculizumab), CD19 (inebilizumab), and the interleukin-6 receptor (satralizumab) have demonstrated efficacy in NMOSD attack prevention in recent phase 3 trials and have gained subsequent regulatory approval in the USA and other countries. We aim to review the evidence supporting the efficacy of these new drugs.

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Authors and Affiliations

  1. Department of Neurology, Mayo Clinic, Rochester, MN, USA

    Cristina Valencia-Sanchez

  2. Department of Neurology, Mayo Clinic, 13400 East Shea Boulevard, Scottsdale, AZ, 85259, USA

    Dean M. Wingerchuk

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  1. Cristina Valencia-Sanchez
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  2. Dean M. Wingerchuk
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Correspondence to Dean M. Wingerchuk.

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Cristina Valencia-Sanchez reports no disclosures. Dean M. Wingerchuk has consulted for MedImmune, Novartis, Biogen, Celgene, Genentech, TG Therapeutics, Third Rock Ventures, Reistone, and Viela Bio. He has received research grants from Alexion and TerumoBCT.

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CV-S drafted the review. DMW critically reviewed and edited the review.

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Valencia-Sanchez, C., Wingerchuk, D.M. Emerging Targeted Therapies for Neuromyelitis Optica Spectrum Disorders. BioDrugs 35, 7–17 (2021). https://doi.org/10.1007/s40259-020-00460-9

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