Promising Breakthrough for Efgartigimod Efficacy in Chronic Immune Thrombocytopenia

Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial

Efgartigimod, a novel therapy for primary immune thrombocytopenia (ITP), has shown significant efficacy in a phase 3 study. ITP, an autoimmune disorder causing low platelet counts and bleeding, was treated with efgartigimod in a 24-week study involving 131 patients. Results revealed a notable increase in sustained platelet count responses, especially in chronic ITP cases. The therapy was well-tolerated, with mild to moderate side effects. This promising outcome suggests a potential breakthrough in ITP treatment.

https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)01460-5/fulltext