FDA Approves New Treatment for Myasthenia Gravis | FDA   
https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-myasthenia-gravis Administration today approved Vyvgart (efgartigimod) for the treatment of generalized

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Quantitative evaluation of drug efficacy in the treatment of myasthenia gravis.

Chen R, Zhang N, Gao L, Zhong Y, Xu L, Liu H, Zheng Q, Li L. Expert Opin Investig Drugs. 2021 Dec 6:1-10. doi: 10.1080/13543784.2021.2010704. Online ahead of print. PMID: 34821184

OBJECTIVES: This study aimed to quantitatively evaluate placebo effect and drug efficacy characteristics and identify associated factors that affect quantitative myasthenia gravis (MG) score (QMGs) and MG activities of daily living score (MG-ADLs) in patients with MG. ...R …

2 Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial.

Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9. PMID: 34146511 Clinical Trial.

We aimed to assess the safety and efficacy of efgartigimod (ARGX-113), a human IgG1 antibody Fc fragment engineered to reduce pathogenic IgG autoantibody levels, in patients with generalised myasthenia gravis. ...Three patients in each treatment group (4%) discontin …

3 The importance of FcRn in neuro-immunotherapies: From IgG catabolism, FCGRT gene polymorphisms, IVIg dosing and efficiency to specific FcRn inhibitors.

Dalakas MC, Spaeth PJ. Ther Adv Neurol Disord. 2021 Feb 26;14:1756286421997381. doi: 10.1177/1756286421997381. eCollection 2021. PMID: 33717213 Free PMC article. Review.

FcRn plays a role in the function of IVIg because the supraphysiological IgG levels derived from IVIg administrations saturate the FcRn allowing the endogenous IgG to be degraded, instead of being recycled, resulting in high levels of infused IgG ensuring IVIg efficiency. New dat …

4 Update in immunosuppressive therapy of myasthenia gravis.

Lascano AM, Lalive PH. Autoimmun Rev. 2021 Jan;20(1):102712. doi: 10.1016/j.autrev.2020.102712. Epub 2020 Nov 13. PMID: 33197578 Free article. Review.

Myasthenia gravis (MG) is an autoimmune disease of the neuromuscular junction. ...

5 Progress in the therapy of myasthenia gravis: getting closer to effective targeted immunotherapies.

Dalakas MC. Curr Opin Neurol. 2020 Oct;33(5):545-552. doi: 10.1097/WCO.0000000000000858. PMID: 32833750 Review.

Three new biologic agents showed promising results in phase-II controlled myasthenia gravis trials: Zilucoplan, a subcutaneous macrocyclic peptide inhibiting complement C5; Efgartigimod, an IgG1-derived Fc fragment binding to neonatal FcRn receptor; and Rozanolixizu …

6 Newer Immunotherapies for the Treatment of Acute Neuromuscular Disease in the Critical Care Unit.

Patel A, Lynch F, Shepherd SA. Curr Treat Options Neurol. 2020 Feb 13;22(3):7. doi: 10.1007/s11940-020-0616-8. PMID: 32052202 Review.

We will discuss epidemiology, pathophysiology, and acute and chronic treatment options for myasthenia gravis, Guillain-Barre syndrome, West Nile virus, Botulism, and amyotrophic lateral sclerosis. ...Efgartigimod showed great promise in a phase 2 safety and efficacy …

7 Current pharmacotherapeutic options for myasthenia gravis.

Barnett C, Tabasinejad R, Bril V. Expert Opin Pharmacother. 2019 Dec;20(18):2295-2303. doi: 10.1080/14656566.2019.1682548. Epub 2019 Oct 31. PMID: 31670984 Review.

Introduction: Novel options for immune-based therapy in myasthenia gravis are improving the therapeutic outlook for patients. ...Further, as immune therapies expand in other areas of medicine, such as oncology, iatrogenic myasthenia is being observed as a complicati …

8 Immunosuppressive and immunomodulatory therapies for neuromuscular diseases. Part II: New and novel agents.

Farmakidis C, Dimachkie MM, Pasnoor M, Barohn RJ. Muscle Nerve. 2020 Jan;61(1):17-25. doi: 10.1002/mus.26711. Epub 2019 Nov 4. PMID: 31531874 Review.

This second article focuses on new and novel immune-based therapies, including eculizumab, a complement inhibitor approved for acetylcholine receptor antibody-positive myasthenia gravis; rituximab, a B-cell depletion therapy with evolving indications in neuromuscular disea …

9 Next-generation Fc receptor-targeting biologics for autoimmune diseases.

Zuercher AW, Spirig R, Baz Morelli A, Rowe T, Käsermann F. Autoimmun Rev. 2019 Oct;18(10):102366. doi: 10.1016/j.autrev.2019.102366. Epub 2019 Aug 9. PMID: 31404703 Free article. Review.

Neonatal Fc receptor (FcRn)-targeting therapeutics block the FcRn receptor and are represented by candidate drugs such as the Fc fragment efgartigimod and the monoclonal antibodies rozanolixizumab (UCB7665), M281 and SYNT001. ...The developmental status of these three clas …

10 Diagnosis and treatment of myasthenia gravis.

Mantegazza R, Cavalcante P. Curr Opin Rheumatol. 2019 Nov;31(6):623-633. doi: 10.1097/BOR.0000000000000647. PMID: 31385879 Review.

PURPOSE OF REVIEW: This article provides an update on the most recent advances in diagnostic procedures and therapeutic approaches for myasthenia gravis, spanning from autoantibody and neuroelectrophysiological tests as diagnostic tools, to innovative and promising treatme …

11 Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis.

Howard JF Jr, Bril V, Burns TM, Mantegazza R, Bilinska M, Szczudlik A, Beydoun S, Garrido FJRR, Piehl F, Rottoli M, Van Damme P, Vu T, Evoli A, Freimer M, Mozaffar T, Ward ES, Dreier T, Ulrichts P, Verschueren K, Guglietta A, de Haard H, Leupin N, Verschuuren JJGM; Efgartigimod MG Study Group. Neurology. 2019 Jun 4;92(23):e2661-e2673. doi: 10.1212/WNL.0000000000007600. Epub 2019 May 22. PMID: 31118245 Free PMC article. Clinical Trial.

OBJECTIVE: To investigate safety and explore efficacy of efgartigimod (ARGX-113), an anti-neonatal Fc receptor immunoglobulin G1 Fc fragment, in patients with generalized myasthenia gravis (gMG) with a history of anti-acetylcholine receptor (AChR) autoantibodies, wh …

12 Efgartigimod: A novel antibody depletion therapy in myasthenia gravis.

Guidon AC, Juel VC. Neurology. 2019 Jun 4;92(23):1079-1080. doi: 10.1212/WNL.0000000000007605. Epub 2019 May 22. PMID: 31118243 No abstract available.

13 Efgartigimod improves muscle weakness in a mouse model for muscle-specific kinase myasthenia gravis.

Huijbers MG, Plomp JJ, van Es IE, Fillié-Grijpma YE, Kamar-Al Majidi S, Ulrichts P, de Haard H, Hofman E, van der Maarel SM, Verschuuren JJ. Exp Neurol. 2019 Jul;317:133-143. doi: 10.1016/j.expneurol.2019.03.001. Epub 2019 Mar 6. PMID: 30851266 Free article.

In MuSK MG, IgG4 anti-MuSK titres closely correlate with disease severity. We therefore tested efgartigimod (ARGX-113), a new neonatal Fc receptor blocker, in a mouse model for MuSK myasthenia gravis. ...These synaptic improvements may well become more explicit upon …

14 Immunotherapy in myasthenia gravis in the era of biologics.

Dalakas MC. Nat Rev Neurol. 2019 Feb;15(2):113-124. doi: 10.1038/s41582-018-0110-z. PMID: 30573759 Review.

No consensus has been reached on the ideal therapeutic algorithm for myasthenia gravis (MG). Most patients with MG require induction therapy with high doses of corticosteroids and maintenance with an immunosuppressant. ...Belimumab, an antibody against the B cell trophic f …