The neonatal Fc receptor inhibitor conferred a modest, short-term benefit for patients with chronic ITP, including those who had received multiple ITP therapies.
Efgartigimod is an investigational, neonatal Fc antibody fragment that targets the neonatal Fc receptor and competitively inhibits binding and, consequently, recycling of IgG antibodies (both pathogenic and nonpathogenic), leading to a significant reduction in IgG antibody half-life. IgG autoantibodies against platelet surface glycoproteins are a main driver of immune thrombocytopenic purpura (ITP) pathogenesis. In a prior phase 2 study, efgartigimod was associated with a clinically relevant improvement in platelet counts versus placebo in patients with ITP (Am J Hematol 2020; 95:178).
Now, investigators report an industry-funded, international, phase 3 study (ADVANCE IV) of efgartigimod in 131 patients with ITP. At baseline, patients had a …
Reviewing Author
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute