[Pubmed] Open-label study of efgartigimod in seronegative myasthenia gravis

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Ther Adv Neurol Disord. 2025 Oct 31;18:17562864251388019. doi: 10.1177/17562864251388019. eCollection 2025.

ABSTRACT

BACKGROUND: The ADAPT trial demonstrated the benefit of efgartigimod, a neonatal Fc receptor (FcRn) inhibitor, in acetylcholine receptor antibody (AChRAb) positive patients with generalized myasthenia gravis (MG). Information regarding the benefits in those lacking pathogenic antibodies is sparse.

OBJECTIVES: We aimed to investigate the safety and efficacy of efgartigimod in patients with double-seronegative (SN) generalized MG.

DESIGN: An open-label 6-month prospective study, conducted at our center.

METHODS: Patients aged at least 18 years with clinical and electrodiagnostic features of MG and negative results for AChRAb and muscle-specific tyrosine kinase antibodies were included. Efgartigimod was administered weekly for 4 weeks and then biweekly for 5 months followed by an observation period. The primary endpoint was the change in MG impairment index (MGII) at 6 months compared to baseline. Secondary endpoints include the change in MG activities of daily living (MG-ADL), other MG scores, overall responders, and early responders. The safety analysis included all patients who received at least one dose of efgartigimod.

RESULTS: We enrolled 30 patients with SN MG who were resistant to other treatments and had unacceptable MGII scores. The MGII decreased by 11.92 points (p < 0.01) with efgartigimod treatment. The MG-ADL also improved. Seventy-two percent of patients were responders with 31% being early responders. Adverse events were reported in 83.3% of patients, and in 90.6%, they were mild. Headache was the most common, reported in 26.7%, followed by flu/common cold in 20%, and urinary tract infection in 13.3%.

CONCLUSION: Efgartigimod was well tolerated and efficacious in patients with SN MG. Future randomized, placebo-controlled studies are needed.

TRIAL REGISTRATION: This trial is registered at ClinicalTrials.gov (NCT06587867), accessed via https://clinicaltrials.gov/study/NCT065 ... mod&rank=1.

PMID:41180124 | PMC:PMC12579174 | DOI:10.1177/17562864251388019


Source: https://pubmed.ncbi.nlm.nih.gov/4118012 ... 22+67771e2