Prior to February 2022, there were no approved treatments for those with cold agglutinin disease (CAD). However, that changed on February 4th, 2022, when the FDA announced that it had approved sutimlimab. According to the American Journal of Managed Care (AJMC), the biologic will be marketed as Enjaymo.

Approving Enjaymo (sutimlimab)

Enjaymo is designed to be administered via intravenous infusion. Prior to its approval, Enjaymo received both Orphan Drug and Breakthrough Therapy designations from the FDA. This humanized monoclonal antibody works by targeting C1s, a protein which plays a role in the activation of the classic complement pathway. Since this pathway often leads to hemolysis (red blood cell death), inhibiting the pathway could benefit patients. In addition to conferring protecting for red blood cells, Enjaymo also reduces the transfusion burden for those with cold agglutinin disease. To learn more about Enjaymo, you can take a look at the full prescribing information provided by the FDA.

Data from the Phase 3 CARDINAL clinical trial ultimately helped this treatment to be approved. During the trial, researchers evaluated the safety, efficacy, and tolerability of sutimlimab for cold agglutinin disease. Altogether, 24 patients enrolled. The trial found that the treatment was effective in increasing hemoglobin levels, reducing the need for blood transfusions, and reducing bilirubin levels. While the therapy was relatively safe and well-tolerated, some side effects did occur. These include arthritis, joint pain, cough, diarrhea, and indigestion.

About Cold Agglutinin Disease (CAD)

Cold agglutinin disease (CAD) is a rare autoimmune disorder. More specifically, CAD is a form of autoimmune hemolytic anemia. In cold agglutinin disease, the immune system mistakenly attacks and destroys red blood cells. This condition may be primary (occurring spontaneously) or secondary (resulting from another condition like mycoplasma pneumonia). Typically, CAD often manifests between ages 40 to 80. It is believed to affect more females than males.

So why is it called “cold” agglutinin disease? Well, CAD symptoms are triggered by cold temperatures. You see, as temperatures drop from 32-50 degrees Fahrenheit, antibodies called cold agglutinins attack and destroy red blood cells. Because of this, symptoms associated with this condition often worsen during winter. These symptoms may include:

• Hemolytic anemia (low red blood cell count and the destruction of red blood cells)
• Fatigue
• Pallor (extremely pale skin)
• Jaundice (yellowing of the skin, eyes, and mucous membranes)
• Dizziness
• Cold hands and feet
• Dark urine
• Headache
• Chest, back, or leg pain
• Vomiting and/or diarrhea
• Heart palpitations
• Skin discoloration