Sutimlimab-jome (Enjaymo®), the only available hemolysis treatment for adults with cold agglutinin disease (CAD), has received an additional indication from the US Food and Drug Administration (FDA) for use in patients without a history of blood transfusions.

The humanized monoclonal antibody, which is designed to selectively target and inhibit the classical complement pathway-specific serine protease C1s, was approved by the FDA in February 2022 for patients with CAD and a recent blood transfusion.

The label expansion is based primarily on the phase 3 CADENZA study, a double-blind, placebo-controlled clinical trial of adults with CAD who did not receive any blood transfusions during the last 6 months.  

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In that study, the use of sutimlimab resulted in hemolysis inhibition as well as avoidance of transfusions and other CAD-related treatments. Moreover, the patients’ hemoglobin levels increased, while their bilirubin and lactate dehydrogenase levels dropped.

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The study participants also reported a statistically significant improvement in symptoms and impacts of fatigue after receiving sutimlimab, as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. The estimated safety profile of sutimlimab from the CARDINAL study was confirmed in phase 3 of the CADENZA study.

“As a first-in-class therapy, Enjaymo can help people living with cold agglutinin disease better manage their symptoms, including the impact of fatigue, which may positively affect their daily life,” noted Bill Sibold, executive vice president and head of specialty care at Sanofi, which markets the treatment.

The most common adverse effects associated with the use of sutimlimab in the studies were rhinitis, cough, headache, hypertension, fatigue, dizziness, nausea, acrocyanosis, Raynaud’s phenomenon, peripheral edema, arthralgia, urinary tract infection, respiratory tract infection, and bacterial infection.

Under the expanded label, the medication will also be available in an undiluted preparation which can reduce the biweekly infusion time to 1 hour.


FDA approves expanded label of Enjaymo® (sutimlimab-jome) to include long-term safety and efficacy for people with cold agglutinin disease. News release. Sanofi; January 25, 2023.