Efgartigimod provided rapid, meaningful symptom relief with few side effects in patients with generalized myasthenia gravis (MG) who did not respond to or could not tolerate standard intravenous immunoglobulin, according to a small real-world study published recently in Neurology and Therapy.
The researchers evaluated the treatment in 13 patients with acetylcholine receptor-positive generalized MG. Patients received four weekly intravenous infusions of efgartigimod at 10 mg/kg after prior treatment with intravenous immunoglobulin. Many of the patients had severe disease, with 70% classified as MGFA class III and 23% as class IVB.
“[It] is important to emphasize that the lack of clinical response to immunoglobulin treatment is not a predictor of the effectiveness of [efgartigimod],” the study’s authors explained.
After just one treatment cycle, all patients experienced clinical improvement. Scores on two standard measures of disease severity improved significantly after four weeks. The Myasthenia Gravis Activities of Daily Living scale dropped by an average of four points, while the Quantitative Myasthenia Gravis score improved by 3.5 points. Response rates were high, with 84.6% of patients achieving meaningful improvement on daily function and 69.2% improving on physician-assessed strength testing.
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Importantly for patients, these benefits were seen regardless of age, sex, disease severity or presence of thymoma or other health conditions. One patient reached minimal symptom expression, meaning symptoms were nearly absent. Improvements were also seen in a patient experiencing myasthenic crisis, including better breathing-related scores.
The treatment also helped reduce reliance on steroids. About 30.7% of patients were able to lower their prednisone dose, with an average reduction of about 6.9 mg. This matters because long-term steroid use can cause significant side effects.
Over a median follow-up of 9.4 months, most patients maintained their improvement, with 72.7% continuing to respond based on symptom scores.
Safety findings were reassuring. Nearly all patients, 92.3%, reported no adverse events. One patient experienced hives and swelling. No infections were observed, even though many patients had severe disease and 84% had other medical conditions.
For patients, these results suggest a promising alternative when standard therapies fail or are poorly tolerated. Efgartigimod works by lowering harmful IgG antibodies that attack the neuromuscular junction, addressing the underlying cause of the disease. While the study was small and retrospective, it reflects real-world care and suggests that this therapy may offer faster symptom relief, fewer side effects and reduced steroid dependence in a population with limited options.
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