Safety and effectiveness of efgartigimod for intravenous infusion in patients with generalized myasthenia gravis: an interim analysis of Japanese post-marketing surveillance.
IF 3.6 3区 医学Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
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Abstract
Background: Efgartigimod for intravenous infusion (efgartigimod-IV) is approved in Japan for generalized myasthenia gravis (gMG). Post-marketing surveillance was mandated by regulatory authorities to assess the safety and effectiveness of efgartigimod in patients with gMG.
Research design and methods: Patients with gMG who received efgartigimod-IV between May 2022 and September 2023 were registered. The interim analysis data cutoff point was June 2024 and included patients whose institutions agreed to publication.
Results: The safety analysis set consisted of 373 patients: 53.35% (n = 199) anti-acetylcholine receptor antibody positive, 14.21% (n = 53) anti-muscle-specific receptor kinase antibody positive, and 31.64% (n = 118) double-seronegative. Adverse drug reaction and serious adverse drug reaction were reported in 21.45% (80/373) and 4.29% (16/373) of patients, respectively. Although six deaths were reported, none of them were related to efgartigimod. The effectiveness analysis set consisted of 246 patients. After three weeks from the first administration, mean score of MG-Activities of Daily Living decreased from 7.5 to 4.4: -3.1 points improvement (standard deviation: 2.95, p < 0.001). No remarkable differences were observed in the response to efgartigimod between the subgroups of patient baseline characteristics, e.g. autoantibody profiles.
Conclusions: In real-world settings, efgartigimod-IV was well tolerated and effective in patients with gMG.
期刊介绍：
Expert Opinion on Biological Therapy (1471-2598; 1744-7682) is a MEDLINE-indexed, international journal publishing peer-reviewed research across all aspects of biological therapy.
Each article is structured to incorporate the author’s own expert opinion on the impact of the topic on research and clinical practice and the scope for future development.
The audience consists of scientists and managers in the healthcare and biopharmaceutical industries and others closely involved in the development and application of biological therapies for the treatment of human disease.
The journal welcomes:
Reviews covering therapeutic antibodies and vaccines, peptides and proteins, gene therapies and gene transfer technologies, cell-based therapies and regenerative medicine
Drug evaluations reviewing the clinical data on a particular biological agent
Original research papers reporting the results of clinical investigations on biological agents and biotherapeutic-based studies with a strong link to clinical practice
Comprehensive coverage in each review is complemented by the unique Expert Collection format and includes the following sections:
Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results;
Article Highlights – an executive summary of the author’s most critical points.
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