Current and emerging biologics for the treatment of neuromyelitis optica spectrum disorders

  title={Current and emerging biologics for the treatment of neuromyelitis optica spectrum disorders},
  author={Ankelien Duchow and Friedemann Paul and Judith Bellmann-Strobl},
  journal={Expert Opinion on Biological Therapy},
  pages={1061 - 1072}
ABSTRACT Introduction Treatment options for patients suffering from neuromyelitis optica spectrum disorders (NMOSD) so far have relied on off-label and empiric drugs. The first drug for the therapy of anti-aquaporin-4 (AQP4) antibody-seropositive NMOSD patients has been approved in 2019: the C5 complement inhibitor eculizumab. The interleukin-6 receptor inhibitor satralizumab and anti-CD19 antibody inebilizumab have published positive phase III trial results and await approval in the near… Expand
Satralizumab in the treatment of neuromyelitis optica spectrum disorder.
Two randomized, double-blind, Phase III trials that investigated the subcutaneous administration of satralizumab as add-on treatment and monotherapy revealed positive effects concerning the reduction of relapse risk for AQP4 seropositive NMOSD patients and generally good tolerability. Expand
Effect of rituximab on disease activity in latin American patients with anti-aquaporin-4 (+) neuromyelitis optica spectrum disorder
These findings support the use of rituximab in patients with Neuromyelitis Optica spectrum disorders with positive AQP4-IgG serostatus, and indirectly suggests that its prompt use could modify the course of the disease. Expand
Eculizumab in the treatment of neuromyelitis optica spectrum disorder.
Eculizumab (Soliris®) was the first drug to be formally approved for the treatment of anti-AQP4-antibody positive NMOSD in 2019. Expand
Rescue immunoadsorption treatment for neuromyelitis optica spectrum disorder attacks unresponsive to intravenous methylprednisolone
IA treatment for IVMP-resistant NMOSD attacks was effective and comparable to PE treatment, and the effective period for IA treatment may be longer than previously thought. Expand
Visualizing the Central Nervous System: Imaging Tools for Multiple Sclerosis and Neuromyelitis Optica Spectrum Disorders
The current status of imaging research in MS and NMOSD is reviewed with an emphasis on emerging modalities that have the potential to be implemented in clinical practice. Expand
Neuromyelitis optica spectrum disorder and myelin oligodendrocyte glycoprotein IgG associated disorder: A comprehensive neuro‐ophthalmic review
The discovery of aquaporin 4 (AQP4) immunoglobulin G (IgG) has improved the ability to diagnose NMOSD and MOGAD, a distinct nosologic entity, which has been more recently described, is considered to have fewer relapses and better prognosis thanNMOSD. Expand
Pain in NMOSD and MOGAD: A Systematic Literature Review of Pathophysiology, Symptoms, and Current Treatment Strategies
A systematic review of the current literature pertaining to pain in NMOSD and MOGAD, focusing on the etiology of their respective pain syndromes and their pathophysiological background is provided. Expand
Complement in neurological disorders and emerging complement-targeted therapeutics
The complement system, the role it plays in autoimmune neurological disease and neurodegenerative disease, and an overview of the latest therapeutics that target complement and that can be used for or have potential in neurological disorders are discussed. Expand
Recent progress in maintenance treatment of neuromyelitis optica spectrum disorder
In NMOSD patients with antibodies against aquaporin 4, monoclonal antibodies that deplete B cells (rituximab and inebilizumab) or interfere with interleukin 6 signaling or complement activation (eculizumAB) have superior efficacy compared to placebo. Expand


Investigational drugs in development to prevent neuromyelitis optica relapses
Eculizumab, a C5 complement inhibitor, may prove useful in the treatment of intractable cases of NMOSD, but physicians must be aware of the known risk of meningococcal infection. Expand
Pharmacotherapy for Neuromyelitis Optica Spectrum Disorders: Current Management and Future Options
The rationale for existing therapeutics and their benefit/risk ratio are described, and the pharmacological and clinical interest of future approaches targeting, among others, B or T cells, the blood–central nervous system barrier, complement, polynuclear cells, AQP4-antibody linkage and AQP 4 activity are discussed. Expand
Challenges and opportunities in designing clinical trials for neuromyelitis optica
This work proposes strategies for NMO clinical trials to evaluate agents targeting recovery from acute attacks and prevention of relapses, the 2 primary goals of NMO treatment. Expand
Long-term Therapy With Interleukin 6 Receptor Blockade in Highly Active Neuromyelitis Optica Spectrum Disorder.
Prolonged tocilizumab therapy may be safe and effective from early treatment phases onward for otherwise therapy-resistant highly active NMO and NMO spectrum disorder. Expand
Neuromyelitis optica spectrum disorders: still evolving and broadening
The clinical, pathological and therapeutic concepts of NMOSD have evolved and broadened over the last two decades following the detection of AQP4 antibodies and MOG antibodies in the patients. Expand
Trial of Satralizumab in Neuromyelitis Optica Spectrum Disorder.
Among patients with NMOSD, satralizumab added to immunosuppressant treatment led to a lower risk of relapse than placebo but did not differ from placebo in its effect on pain or fatigue. Expand
Failure of natalizumab to prevent relapses in neuromyelitis optica.
It is suggested that Neuromyelitis optica should be considered as a differential diagnosis in patients with suspected MS who are unresponsive to natalizumab therapy. Expand
Efficacy and Safety of Rituximab Therapy in Neuromyelitis Optica Spectrum Disorders: A Systematic Review and Meta-analysis.
Evidence is provided that rituximab therapy reduces the frequency of NMOSD relapses and neurological disability in patients with NMOSDs, however, the safety profile suggests caution in prescribing ritUXimab as a first-line therapy. Expand
Combination of cyclosporine A with corticosteroids is effective for the treatment of neuromyelitis optica
CyA as well as AZA may help stabilize the disease activity in NMO/NMOSD patients seropositive for anti-AQP4 antibodies, and this is the first case series study demonstrating the efficacy of CyA for the treatment of NMO /NMOSDs. Expand
Methotrexate is an alternative to azathioprine in neuromyelitis optica spectrum disorders with aquaporin-4 antibodies
Methotrexate is a commonly prescribed drug in general practice and when used in NMO it reduces relapse frequency, stabilises disability and is well tolerated, even in patients who have failed one or more other treatments. Expand