Understanding Cold Agglutinin Disease
Cold agglutinin disease (CAD) is a severe, chronic, rare blood disorder.1 It is a type of autoimmune hemolytic anemia (AIHA), which means that the body’s immune system attacks and destroys its own red blood cells by mistake.2
Symptoms of CAD may include fatigue, dizziness, chest pain, shortness of breath, anemia, transfusion requirements, dark urine and increased risk of thrombotic events like stroke or heart attack.2
In people with CAD, autoantibodies such as immunoglobin M (IgM) cause red blood cells to clump together when extremities (nose, ears, fingers, toes) are exposed to cold temperatures or when an individual with CAD has a compromised immune system or infection.3 Then, the complement cascade, which plays an important role in the immune system, is activated to destroy heathy red blood cells in a process called hemolysis.4,5
Excessive activation of the complement cascade can lead to the onset or progression of many diseases including CAD.6,7
There are no approved medicines available for the treatment of CAD, so there is a significant unmet need for new therapies.
Pegcetacoplan (APL-2) in CAD
Pegcetacoplan is an investigational targeted C3 inhibitor designed to regulate uncontrolled or excessive complement activation. We believe this approach may stop the body from attacking its own red blood cells and minimize the symptoms associated with CAD.
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