FDA approves sutimlimab for cold agglutinin disease

The U.S. Food and Drug Administration (FDA) has approved sutimlimab-jome (Enjaymo; Sanofi) to decrease the need for red blood cell transfusion due to hemolysis in adults with cold agglutinin disease (CAD), a rare type of anemia, according to a company press release. This is the first approved treatment for CAD.

Approval is based on results from a 26-week open label, single arm pivotal Phase 3 study. In the CARDINAL study, 24 patients with CAD and a recent history of blood transfusion were treated with sutimlimab. Overall, 54% of patients met the composite primary endpoint criteria with 63% of patients achieving a hemoglobin ≥ 12 g/dL or an increase of ≥2 g/dL.

After week 5, 71% of patients were transfusion-free. In 92% of patients, no other CAD-related treatments were used.

The most common adverse reactions were respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral edema. Serious adverse reactions included streptococcal sepsis and staphylococcal wound infection (n = 1), arthralgia (n = 1), and respiratory tract infection (n = 1). None of the adverse reactions led to discontinuation of treatment in the study; dosage interruptions due to an adverse reaction occurred in 17% of patients.

“For people living with cold agglutinin disease, it is as if their body’s immune system is waging a war on itself. The relentless destruction of healthy red blood cells is a daily, silent reality for people with CAD. For the first time, we have a treatment that targets complement-mediated hemolysis, which is the underlying cause of the red blood cell destruction in many CAD patients. In the pivotal study, patients treated with sutimlimab had an improvement in anemia as measured by hemoglobin and bilirubin levels during the 26-week study,” said Catherine Broome, MD,

Associate professor of medicine at Georgetown University Lombardi Comprehensive Cancer Center, and a principal investigator in the CARDINAL study.