Adrian C. Newland, MD, of the Royal London Hospital in London, U.K. provided an update on the Phase 2 clinical trial looking at the safety and efficacy of efgartigimod to treat primary immune thrombocytopenia (ITP).
Results of the Phase 2 study were presented at the American Society of Hematology (ASH) annual meeting in Orlando, FL. In the study, adult ITP patients were randomized to receive 4 weekly intravenous infusions of either placebo or efgartigimod at a dose of 5 mg/kg or 10 mg/kg.
The primary endpoint was safety and secondary endpoints included pharmacodynamic (PD) markers, pharmacokinetic (PK) parameters, presence of anti-drug antibodies (ADA) and autoantibodies, and efficacy. As Dr. Newland explains, efgartigimod was well tolerated with no dose-related safety observations. Furthermore, treatment with efgartigimod resulted in a rapid reduction of total IgG and all IgG subtypes in all treated patients.
Immune thrombocytophenia (ITP) is a bleeding disorder due to an unusually low level of platelets. Affected individuals bleed and bruise easily. Common symptoms include petechiae (red or purple spots on the skin), bleeding from the gums or nose, blood in urine or stool, and/or heavy menstrual bleeding.
For more information about this and other blood disorders, visit https://checkrare.com/diseases/blood-diseases/
