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. 2025 Jul;46(7):3271-3279.
doi: 10.1007/s10072-025-08096-9. Epub 2025 Mar 25.

Efgartigimod improves non-AChR generalized Myasthenia Gravis: a real world experience

Carlo Antozzi  1   2   3 , Rita Frangiamore  4 , Elena Rinaldi  4 , Fiammetta Vanoli  4   5 , Francesca Andreetta  4 , Eleonora Giacopuzzi Grigoli  4 , Emilio Ciusani  6 , Silvia Bonanno  4 , Lorenzo Maggi  4 , Renato Mantegazza  4
Affiliations

Efgartigimod improves non-AChR generalized Myasthenia Gravis: a real world experience

Carlo Antozzi et al. Neurol Sci. 2025 Jul.

Abstract

Introduction: The neonatal Fc receptor (FcRn) inhibitor Efgartigimod (EFG) has been approved for treatment of generalized Myasthenia Gravis (gMG) with anti-AChR antibodies. Information on the effect of EFG in non-AChR MG is limited. We investigated the efficacy of EFG in non-AChR gMG along a clinical follow-up of 2 years.

Methods: We treated 13 patients with gMG without anti-AChR antibodies: 5 MuSK+, 2 LRP4 + and 6 triple-negative (confirmed by live CBA). EFG was administered according to the GENERATIVE protocol (consisting of a Fixed period of 2 treatment cycles of 4 infusions at weekly intervals, followed by a Flexible period during which EFG was given in case of initial worsening) starting from November 2021. Outcomes were evaluated by means of the MG-ADL, QMG, MGC and MGQoL15r scales.

Results: The mean follow-up was 21 ± 5.3 months. Meaningful improvement was observed with the clinical scores adopted. The number of cycles/year was 3.92 ± 0.9. The interval between cycles was 10.1 ± 3.6 weeks. MG-ADL improvement of at least 5 points was recorded in 58% of cycles. 46% of patients required hospitalization during the two years before treament with EFG and 70% plasmaexchange/IVIG; during EFG none of the patients was hospitalized or required immunomodulation. No major side effects or infusion related reactions occurred.

Conclusion: EFG was effective in non-AChR gMG and modified significantly the course of the disease. Our experience strengthens the role of FcRn inhibition as a new therapeutic tool for MG without anti-AChR antibodies.

Keywords: Efgartigimod; FcRn inhibitors; LRP4; MuSK; Myasthenia Gravis.

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Conflict of interest statement

Declarations. Ethical approval: The study was approved by the Institutional Review Board of the local hospital. Competing interests: C. Antozzi received funding for travel, meeting attendance and advisory board participation from Alexion, Momenta, Sanofi, Janssen, argenx and UCB. F. Vanoli received funding for travel, meeting attendance and consulting by Alexion Pharmaceuticals, UCB Pharma and Argenx. S. Bonanno received funding for travel, meeting attendance and advisory board participation from Sanofi Genzyme, Biogen, Alexion and Roche. R. Frangiamore received funding for travel, consulting, meeting attendance and advisory board participation by argenx, UCB, Alexion, Biogen, Sanofi Genzyme. L. Maggi received funding for travel, meeting attendance, honoraria for speaking and advisory board participation from Sanofi Genzyme, Roche, Biogen, Amicus Therapeutics, Alexion, UCB, argenx, Janssen, Lupin. R. Mantegazza received funding for travel, meeting attendance and advisory board participation from Alexion, argenx, Biomarin, Catalyst, Sanofy Genzyme, Regeneron and UCB. E. Rinaldi, F. Andreetta, E. Ciusani and E. Giacopuzzi have nothing to disclose.

References

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