Jul 09, 2025
At EAN 2025, new data highlighted the potential of efgartigimod, a neonatal Fc receptor (FcRn) inhibitor, as an add-on to intravenous methylprednisolone (IVMP) for patients experiencing acute attacks of Neuromyelitis Optica Spectrum Disorder (NMOSD) and Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease (MOGAD)—two rare but debilitating autoimmune demyelinating conditions driven by pathogenic IgG autoantibodies.
In this exploratory study of 27 adult patients (13 on efgartigimod + IVMP; 14 on IVMP alone), efgartigimod was administered intravenously 10mg/kg × 4 doses or 20mg/kg × 2 doses. Adjunctive efgartigimod led to significantly greater neurological improvement, with a mean reduction in Expanded Disability Status Scores (EDSS) of 1.3 ± 0.6 vs. 0.5 ± 0.5 in the control group. Biologically, the therapeutic impact was underscored by a marked 69.8% decrease in total serum IgG and antibody titer reduction in nearly 70% of treated patients—suggesting efgartigimod’s mechanism of reducing circulating pathogenic antibodies translated into meaningful clinical benefit.
Notably, patients receiving the more intensive dosing regimen (20 mg/kg × 2) experienced faster and more pronounced improvements in both EDSS scores and antibody titers. Safety was acceptable, with no new safety signals observed, supporting the tolerability of FcRn blockade in the acute setting.
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These findings reinforce FcRn antagonism as a mechanistically targeted strategy for immunoglobulin-mediated CNS disorders and pave the way for larger, controlled trials to define the role of efgartigimod in both NMOSD and MOGAD relapse management.
KOL Views
The introduction of FcRn blockers like efgartigimod represents a significant advance in the acute management of NMOSD and MOGAD. Early clinical results are encouraging, particularly for patients who do not respond adequately to steroids alone. However, larger controlled studies are required. – Expert Opinion
While efgartigimod and similar biologics are reshaping the treatment landscape for antibody-mediated CNS disorders, careful patient selection and biomarker-driven monitoring will be key. – Expert Opinion
ConclusionEfgartigimod, an FcRn inhibitor, shows substantial promise as an add-on therapy to IVMP for acute NMOSD and MOGAD. Clinical data indicate that efgartigimod leads to a greater reduction in disability (as measured by EDSS), a significant decrease in pathogenic antibody titers, and a rapid decline in serum IgG levels compared to IVMP alone. The therapy is well-tolerated, with no serious adverse events reported in the available studies. However, the current evidence is limited by small sample sizes and short follow-up periods, and larger, randomized controlled trials are needed to confirm both the long-term efficacy and safety of efgartigimod in these patient populations.
Article in PDF
Jul 09, 2025
At EAN 2025, new data highlighted the potential of efgartigimod, a neonatal Fc receptor (FcRn) inhibitor, as an add-on to intravenous methylprednisolone (IVMP) for patients experiencing acute attacks of Neuromyelitis Optica Spectrum Disorder (NMOSD) and Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease (MOGAD)—two rare but debilitating autoimmune demyelinating conditions driven by pathogenic IgG autoantibodies.
In this exploratory study of 27 adult patients (13 on efgartigimod + IVMP; 14 on IVMP alone), efgartigimod was administered intravenously 10mg/kg × 4 doses or 20mg/kg × 2 doses. Adjunctive efgartigimod led to significantly greater neurological improvement, with a mean reduction in Expanded Disability Status Scores (EDSS) of 1.3 ± 0.6 vs. 0.5 ± 0.5 in the control group. Biologically, the therapeutic impact was underscored by a marked 69.8% decrease in total serum IgG and antibody titer reduction in nearly 70% of treated patients—suggesting efgartigimod’s mechanism of reducing circulating pathogenic antibodies translated into meaningful clinical benefit.
Notably, patients receiving the more intensive dosing regimen (20 mg/kg × 2) experienced faster and more pronounced improvements in both EDSS scores and antibody titers. Safety was acceptable, with no new safety signals observed, supporting the tolerability of FcRn blockade in the acute setting.
These findings reinforce FcRn antagonism as a mechanistically targeted strategy for immunoglobulin-mediated CNS disorders and pave the way for larger, controlled trials to define the role of efgartigimod in both NMOSD and MOGAD relapse management.
KOL Views
The introduction of FcRn blockers like efgartigimod represents a significant advance in the acute management of NMOSD and MOGAD. Early clinical results are encouraging, particularly for patients who do not respond adequately to steroids alone. However, larger controlled studies are required. – Expert Opinion
While efgartigimod and similar biologics are reshaping the treatment landscape for antibody-mediated CNS disorders, careful patient selection and biomarker-driven monitoring will be key. – Expert Opinion
ConclusionEfgartigimod, an FcRn inhibitor, shows substantial promise as an add-on therapy to IVMP for acute NMOSD and MOGAD. Clinical data indicate that efgartigimod leads to a greater reduction in disability (as measured by EDSS), a significant decrease in pathogenic antibody titers, and a rapid decline in serum IgG levels compared to IVMP alone. The therapy is well-tolerated, with no serious adverse events reported in the available studies. However, the current evidence is limited by small sample sizes and short follow-up periods, and larger, randomized controlled trials are needed to confirm both the long-term efficacy and safety of efgartigimod in these patient populations.
