Emerging drugs for the treatment of neuromyelitis optica

ABSTRACT

Introduction

Evidence-based treatment options for neuromyelitis optica spectrum disorders (NMOSD) patients are beginning to enter the market. Where previously, there was only the exclusive use of empiric and off-label immunosuppressants in this rare and devastating central nervous system autoimmune disease.

Areas covered

In accordance to expanding pathogenetic insights, drugs in phase II and III clinical trials are presented in the context of the current treatment situation for acute attacks and immunopreventative strategies in NMOSD. Some such drugs are the 2019-approved complement inhibitor eculizumab, other compounds in late development include its modified successor ravulizumab, IL-6 receptor antibody satralizumab, CD19 targeting antibody inebilizumab and the TACI-Fc fusion protein telitacicept.

Expert opinion

Moving from broad immunosuppression to tailored treatment strategies, the prospects for efficient NMOSD therapy are positive. For the first time in this disease, class I treatment evidence is available, but long-term data will be necessary to confirm the overall promising study results of the compounds close to approval. While drug development still centers around AQP4 antibody seropositive patients, current and future research requires consideration of possible diverging treatment demands for the smaller group of seronegative patients and patients with presence of MOG antibodies.

KEYWORDS:

• Neuromyelitis Optica Spectrum Disorders
• antibody therapy
• eculizumab
• satralizumab
• inebilizumab
• telitacicept
• ravulizumab

Declaration of interest

A Duchow has received a speaker honorarium from Roche, not related to the preparation of this manuscript. C Chien has nothing to disclose. F Paul has served on the scientific advisory boards of Novartis and MedImmune; received speaker honoraria and travel funding from Bayer, Novartis, Biogen, Teva, Sanofi-Aventis/Genzyme, Merck Serono, Alexion, Chugai, MedImmune, and Shire; serves as academic editor of PLoS ONE and associate editor of Neurology: Neuroimmunology & Neuroinflammation; consulted for Sanofi-Genzyme, Biogen, MedImmune, Shire, and Alexion; and received research support from Bayer, Novartis, Biogen, Teva, Sanofi-Aventis/Genzyme, Alexion, Merck Serono, German Research Council, Werth Stiftung of the City of Cologne, German Ministry of Education and Research, Arthur Arnstein Stiftung Berlin, EU FP7 Framework Program, Guthy Jackson Charitable Foundation, and NMSS. J Bellmann-Strobl has received travel grants and speaking fees from Bayer Healthcare, Biogen Idec, Merck Serono, Roche, Sanofi Genzyme, Teva Pharmaceuticals, and Novartis, none of them related to preparing this manuscript. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

A reviewer on this manuscript has received personal compensation for consulting, serving on a scientific advisory board, speaking, professional travel/accommodation stipends or other activities with Biogen-Idec, Genzyme, Merck-Serono, Novartis, Teva, Roche and Bayer. In addition, they have received professional travel/accommodation stipends from Guthy-Jackson Charitable Foundation. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.