584
Views
1
CrossRef citations to date
0
Altmetric
Drug Profile

Efgartigimod for generalized myasthenia gravis with or without anti-acetylcholine receptor antibodies: a worldwide and Japanese perspective

ORCID Icon, &
Pages 1207-1215 | Received 31 Aug 2022, Accepted 11 Oct 2022, Published online: 17 Oct 2022

References

  • Punga AR, Maddison P, Heckmann JM, et al. Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176–188.
  • Klein CJ, Beecher G, Lamb C, et al. LRP4-IgG service line testing in seronegative myasthenia gravis and controls. J Neuroimmunol. 2022;368:577895.
  • Burns TM, Sadjadi R, Utsugisawa K, et al. International clinimetric evaluation of the MG-QOL15, resulting in slight revision and subsequent validation of the MG-QOL15r. Muscle Nerve. 2016;54(6):1015–1022.
  • Murai H. Japanese clinical guidelines for myasthenia gravis: putting into practice. Clin Exp Neuroimmunol. 2015;6(1):21–31.
  • Utsugisawa K, Suzuki S, Nagane Y, et al. Health-related quality-of-life and treatment targets in myasthenia gravis. Muscle Nerve. 2014;50(4):493–500.
  • Murai H, Utsugisawa K, Nagane Y, et al. Rationale for the clinical guidelines for myasthenia gravis in Japan. Ann N Y Acad Sci. 2018;1413(1):35–40.
  • Masuda M, Utsugisawa K, Suzuki S, et al. The MG-QOL15 Japanese version: validation and associations with clinical factors. Muscle Nerve. 2012;46(2):166–173.
  • Jaretzki A 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. task force of the medical scientific advisory board of the Myasthenia Gravis Foundation of America. Neurology. 2000;55(1):16–23.
  • Narayanaswami P, Sanders DB, Wolfe G, et al. International consensus guidance for management of myasthenia gravis: 2020 update. Neurology. 2021;96(3):114–122.
  • Lascano AM, Lalive PH. Update in immunosuppressive therapy of myasthenia gravis. Autoimmun Rev. 2021;20(1):102712.
  • Vanoli F, Mantegazza R. Antibody therapies in autoimmune neuromuscular junction disorders: approach to myasthenic crisis and chronic management. Neurotherapeutics. 2022;19(3):897–910.
  • Imai T, Suzuki S, Nagane Y, et al. Reappraisal of oral steroid therapy for myasthenia gravis. Front Neurol. 2020;11:868.
  • Nishida Y, Takahashi YK, Kanai T, et al. Safety of tapering tacrolimus dose in patients with well-controlled anti-acetylcholine receptor antibody-positive myasthenia gravis. Eur J Neurol. 2020;27(1):100–104.
  • Wolfe GI, Kaminski HJ, Aban IB, et al. Randomized trial of thymectomy in myasthenia gravis. N Engl J Med. 2016;375(6):511–522.
  • Grob D, Brunner N, Namba T, et al. Lifetime course of myasthenia gravis. Muscle Nerve. 2008;37(2):141–149.
  • Utsugisawa K, Nagane Y, Uzawa A, et al. Health-related quality of life and treatment strategies in myasthenia gravis. Clin Exp Neuroimmunol. 2020;11(4):209–217.
  • Ozawa Y, Uzawa A, Yasuda M, et al. Long-term outcomes and prognostic factors in generalized myasthenia gravis. J Neurol. 2021;268(10):3781–3788.
  • Nagane Y, Murai H, Imai T, et al. Social disadvantages associated with myasthenia gravis and its treatment: a multicentre cross-sectional study. BMJ open. 2017;7(2):e013278.
  • Tran C, Biswas A, Mendoza M, et al. Performance of different criteria for refractory myasthenia gravis. Eur J Neurol. 2021;28(4):1375–1384.
  • Schneider-Gold C, Hagenacker T, Melzer N, et al. Understanding the burden of refractory myasthenia gravis. Ther Adv Neurol Disord. 2019;12:1756286419832242.
  • Hehir MK, Hobson-Webb LD, Benatar M, et al. Rituximab as treatment for anti-MuSK myasthenia gravis: multicenter blinded prospective review. Neurology. 2017;89(10):1069–1077.
  • Dalakas MC. Role of complement, anti-complement therapeutics, and other targeted immunotherapies in myasthenia gravis. Expert Rev Clin Immunol. 2022;18(7):691–701.
  • Peter HH, Ochs HD, Cunningham-Rundles C, et al. Targeting FcRn for immunomodulation: benefits, risks, and practical considerations. J Allergy Clin Immunol. 2020;146(3):479–91.e5.
  • Brambell FW. The transmission of immunity from mother to young and the catabolism of immunoglobulins. Lancet. 1966;2(7473):1087–1093.
  • Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 2007;7(9):715–725.
  • Ober RJ, Martinez C, Lai X, et al. Exocytosis of IgG as mediated by the receptor, FcRn: an analysis at the single-molecule level. Proc Natl Acad Sci U S A. 2004;101(30):11076–11081.
  • Ulrichts P, Guglietta A, Dreier T, et al., Neonatal Fc receptor antagonist efgartigimod safely and sustainably reduces IgGs in humans. J Clin Invest. 2018;128(10):4372–4386.
  • Huijbers MG, Plomp JJ, van Es IE, et al. Efgartigimod improves muscle weakness in a mouse model for muscle-specific kinase myasthenia gravis. Exp Neurol. 2019;317:133–143.
  • Howard JF Jr., Bril V, Burns TM, et al. Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. Neurology. 2019;92(23):e2661–e73.
  • Howard JF Jr., Bril V, Vu T, et al., Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021;20(7):526–536.
  • Howard JF Jr, Bril V, Vu T, et al. Long-term safety, tolerability, and efficacy of efgartigimod in patients with generalized myasthenia gravis: interim results of the ADAPT+ study. 2022 AAN Annual meeting abstracts by American Academy of Neurology Published on Feb 24. Seattle, Washingtom, 2022.
  • Heo YA. Efgartigimod: first approval. Drugs. 2022;82(3):341–348.
  • Newland AC, Sánchez-González B, Rejtő L, et al. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020;95(2):178–187.
  • Goebeler M, Bata-Csörgő Z, De Simone C, et al. Treatment of pemphigus vulgaris and foliaceus with efgartigimod, a neonatal Fc receptor inhibitor: a phase II multicentre, open-label feasibility trial. Br J Dermatol. 2022;186(3):429–439.
  • Suzuki S. Targeted molecular therapy for myasthenia gravis. Lancet Neurol. 2021;20(7):499–500.
  • Howard JF Jr., Utsugisawa K, Benatar M, et al. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017;16(12):976–986.
  • Mantegazza R, Wolfe GI, Muppidi S, et al. Post-intervention status in patients with refractory myasthenia gravis treated with eculizumab during REGAIN and its open-label extension. Neurology. 2021;96(4):e610–e8.
  • Oyama M, Okada K, Masuda M, et al. Suitable indications of eculizumab for patients with refractory generalized myasthenia gravis. Ther Adv Neurol Disord. 2020;13:1756286420904207.
  • Murai H, Suzuki S, Hasebe M, et al. Safety and effectiveness of eculizumab in Japanese patients with generalized myasthenia gravis: interim analysis of post-marketing surveillance. Ther Adv Neurol Disord. 2021;14:17562864211001995.
  • Vu T, Meisel A, Mantegazza R, et al. Terminal complement inhibitor ravulizumab in generalized myasthenia gravis. NEJM Evid. 2022;1. doi:10.1056/EVIDoa2100066
  • Howard JF Jr., Nowak RJ, Wolfe GI, et al. Clinical effects of the self-administered subcutaneous complement inhibitor zilucoplan in patients with moderate to severe generalized myasthenia gravis: results of a phase 2 randomized, double-blind, placebo-controlled, multicenter clinical trial. JAMA Neurol. 2020;77(5):582–592.
  • Muppidi S, Utsugisawa K, Benatar M, et al. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. Muscle Nerve. 2019;60(1):14–24.
  • Suzuki S, Utsugisawa K, Suzuki N. Overlooked non-motor symptoms in myasthenia gravis. J Neurol Neurosurg Psychiatry. 2013;84(9):989–994.
  • Tomschik M, Hilger E, Rath J, et al. Subgroup stratification and outcome in recently diagnosed generalized myasthenia gravis. Neurology. 2020;95(10):e1426–e36.
  • Bril V, Benatar M, Andersen H, et al. Efficacy and safety of rozanolixizumab in moderate to severe generalized myasthenia gravis: a phase 2 randomized control trial. Neurology. 2021;96(6):e853–e65.
  • Dalakas MC, Spaeth PJ. The importance of FcRn in neuro-immunotherapies: from IgG catabolism, FCGRT gene polymorphisms, IVIg dosing and efficiency to specific FcRn inhibitors. Ther Adv Neurol Disord. 2021;14:1756286421997381.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.