Efgartigimod Discovered as an Effective Drug for Autoimmune Blood Disorders - medtigo



Efgartigimod Discovered as an Effective Drug for Autoimmune Blood Disorders

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According to The Print, the investigational drug efgartigimod is being studied to treat chronic primary immune thrombocytopenia (ITP). Patients who received it saw a significant increase in platelet counts than those who received a placebo, demonstrating the importance of platelets in clotting and bleeding control.

Georgetown University Medical Center participated in the worldwide ADVANCE IV clinical trial. People with ITP have high levels of an autoantibody known as immunoglobulin G (IgG), which causes increased platelet clearance from the blood and, perhaps, decreased platelet synthesis.

ITP can be challenging to treat, especially if the patient has previously failed to react to ITP medicines. The ADVANCE IV research results explore the potential benefits of efgartigimod as a therapy for ITP. According to Broome, there is still a great demand for ITP therapy.

Because ITP is associated with excessive exhaustion and has been demonstrated to hurt mental health (through symptoms such as worry, fear, and depression), finding new treatments for it has been crucial.  

Every year in the United States, around 3,3 new cases of ITP are diagnosed for every 100,000 persons in the general population. Between the ages of 20 and 60, there is a significant gender discrepancy in the incidence of ITP.  

The mechanism of action of efgartigimod is new. It successfully decreased IgG levels without altering lymphocytes, IgG synthesis, or the innate immune system. In the ADVANCE IV research, 131 people from the United States, the European Union, and Japan participated in a randomized, placebo-controlled medication trial.

For 24 weeks, subjects were given either efgartigimod or a placebo. All individuals in the study had low platelet counts and previously had at least one ITP therapy, with the majority having received three or more ITP treatments.  

Argenx, the company that invented efgartigimod, funded the study. Myasthenia gravis is an autoimmune disease characterized by poor nerve and muscle transmission, and Vyvgart is the only brand name approved to treat it.  

Patients with chronic ITP who received efgartigimod exhibited considerably more significant rates of persistent platelet response than those who received a placebo across at least four of the last six planned trial visits, with about half of those who reacted seeing their platelet counts doubled. These findings were discovered in the ADVANCE IV study.

The therapy was beneficial in various patient demographics, with favorable outcomes demonstrated across all ages, illness stages, time since diagnosis, past ITP therapies, and other drugs.


The medication’s most often reported adverse effects were those associated with bleeding, such as bruising, headache, blood in the urine, and rash-like skin symptoms.

Because no serious adverse effects were identified, the treatment looked to have no substantial drawbacks. “We believe that bleeding episodes and fatigue will lessen, resulting in an overall improvement in quality of life,” adds Broome, as new medicines become accessible to patients with ITP.  

The medication was provided through intravenous injection during this research. It is also being tested with IV delivery through the skin. The findings of the subcutaneous study are scheduled to be revealed by the end of 2023. 

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