To synthesize evidence from pivotal trials to real-world applications regarding the efficacy and safety of efgartigimod in treating generalized myasthenia gravis (gMG).
Approach:
Key Findings:
Efgartigimod showed rapid, clinically meaningful improvements in MG-ADL scores in AChR-Ab+ patients.
The phase 3 ADAPT trial demonstrated a significant response rate of 68% in efgartigimod-treated patients compared to 30% in placebo.
Long-term studies confirmed reproducible efficacy and sustained safety across multiple treatment cycles.
Real-world evidence supports rapid response and steroid-sparing effects in various patient subgroups, including antibody-negative and thymoma-associated MG patients.
Interpretation:
Limitations:
Comparative efficacy against other novel biologics is still under investigation.
Long-term effects and optimal patient selection require further research.