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Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future

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Abstract

Myasthenia gravis (MG) is a rare autoimmune disease that causes debilitating muscle weakness due to impaired neuromuscular transmission. Since most (about 80–90%) MG patients present autoantibodies against the acetylcholine receptor, standard medical therapy consists of symptomatic treatment with acetylcholinesterase inhibitors (e.g., pyridostigmine). In addition, considering the autoimmune basis of MG, standard therapy includes immunomodulating agents, such as corticosteroids, azathioprine, cyclosporine A, and cyclophosphamide. New strategies have been proposed for the treatment of MG and include complement blockade (i.e., eculizumab, ravulizumab, and zilucoplan) and neonatal Fc receptor antagonism (i.e., efgartigimod and rozanolixizumab). The aim of this review is to provide a detailed overview of the pre- and post-marketing evidence on the five pharmacological treatments most recently approved for the treatment of MG, by identifying both preclinical and clinical studies registered in clinicaltrials.gov. A description of the molecules currently under evaluation for the treatment of MG is also provided.

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Author notes

  1. Salvatore Crisafulli and Brigida Boccanegra equal contribution as first authors.

  2. Gianluca Trifirò and Annamaria De Luca equal contribution as senior authors.

Authors and Affiliations

  1. Department of Medicine, University of Verona, Verona, Italy

    Salvatore Crisafulli

  2. Department of Pharmacy-Drug Sciences, University of Bari “Aldo Moro”, Bari, Italy

    Brigida Boccanegra, Paola Mantuano & Annamaria De Luca

  3. Department of Diagnostics and Public Health, University of Verona, P.le L.A. Scuro 10, 37124, Verona, Italy

    Massimo Carollo, Emanuela Bottani & Gianluca Trifirò

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  1. Salvatore Crisafulli
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Correspondence to Gianluca Trifirò.

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GT has served in the last 3 years on advisory boards/seminars funded by Sanofi, Eli Lilly, AstraZeneca, Abbvie, Novo Nordisk, Gilead, and Amgen; he is also a scientific coordinator of the academic spin-off “INSPIRE srl”, which has received funding for conducting observational studies from several pharmaceutical companies. None of these listed activities is related to the topic of the article. SC, BB, EB, MC, PM, and ADL 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 article.

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SC: literature search, writing, draft revision; BB: literature search, writing, draft revision; MC: literature search, writing, draft revision; EB: literature search, writing, draft revision; PM: literature search, writing, draft revision; GT: conceptualization, draft revision; ADL: conceptualization, draft revision. All authors have read and approved the final submitted manuscript.

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Crisafulli, S., Boccanegra, B., Carollo, M. et al. Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future. CNS Drugs 38, 15–32 (2024). https://doi.org/10.1007/s40263-023-01059-8

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