Press release: European Commission approves Enjaymo® (sutimlimab) for the treatment of hemolytic anemia in adults with cold agglutinin disease – 11/17/2022 at 6:50 p.m.

The European Commission approves Enjaymo


(sutimlimab) for treatment of hemolytic anemia in adults suffering from cold agglutinin disease

  • Enjaymo is the first and only approved treatment option for the treatment of hemolytic anemia in adults with cold agglutinin disease.

Paris, November 17, 2022

. The European Commission (EC) has granted marketing authorization to Enjaymo


(sutimlimab) for treatment of hemolytic anemia in adults with cold agglutinin disease (CAD), a chronic, rare and severe autoimmune hemolytic anemia that causes the immune system to mistakenly attack healthy red blood cells, causing their degradation or haemolysis.


Dietmar Berger



Chief Medical Officer, Global Head, Development, Sanofi

“This approval illustrates our commitment to developing drugs that are first-in-class and transform patients’ lives. Until today in Europe, patients were forced to avoid exposure to cold, receive blood transfusions and take drugs not indicated for the treatment of FAD to relieve their disease. The European Commission’s approval gives these patients for the first time access to a drug that can significantly transform the management of this disease and their daily lives. »

Enjaymo is currently the only drug approved for the treatment of FAD. It is the first humanized monoclonal antibody in its pharmacotherapeutic class designed to specifically target and inhibit the serine protease specific to the classical complement pathway, the C1s complex. Enjaymo 50mg/ml will be presented as a solution for infusion.


Alexander R



Department of Hematology and Stem Cell Transplantation, University Hospital, University of Duisburg and Essen, Germany

“In addition to the long diagnostic wandering that patients with FAD experience, the impact of fatigue on their quality of life is often debilitating and comparable to that of diseases such as cancer-related anemia and other autoimmune disorders. . Clinicians now have an essential treatment option for their patients. »

About the CADENZA and CARDINAL clinical trials

EC approval is based on data from two Phase III clinical trials: CADENZA, a double-blind, placebo-controlled clinical trial in adults with cold agglutinin disease without a recent history of blood transfusion (in six months), and the CARDINAL trial, a pivotal, 26-week, single-arm, open-label study in patients with FAD who had recently undergone blood transfusion.

In Part A of the CADENZA trial, eligible patients were randomized 1:1 to receive either a fixed dose of Enjaymo calculated by weight (6.5g or 7.5g), or a placebo, by intravenous infusion, on Day 0, Day 7, then every other week for a maximum duration of 26 weeks. The open-label Part B of the trial assessed the long-term safety of Enjaymo, as well as the duration of drug response, in patients with FAD. The primary composite endpoint and all of the secondary endpoints from Part A of the CADENZA study were met and Enjaymo provided inhibition of hemolysis, increase in hemoglobin levels and improvement clinically significant fatigue scores measured using the FACIT scale (

Functional Assessment of Chronic Illness Therapy

, evaluation of the impact of the treatment of chronic diseases). Enjaymo presented an acceptable safety profile and was generally well tolerated. Ninety-six percent (96%) of patients in the Enjaymo group and 100% of patients in the placebo group experienced at least one treatment-related adverse event. The following adverse events were reported more frequently by patients treated with Enjaymo compared to those treated with placebo: headache (22.7% versus 10.0%), high blood pressure (22.7% versus 0%), rhinitis (18.2% versus 0%), Raynaud’s syndrome (18.2% versus 0%) and acrocyanosis (13.6% versus 0%).

In Part A of the CARDINAL trial, patients received a fixed dose of Enjaymo calculated by weight (6.5g or 7.5g) by intravenous infusion on Day 0, Day 7 , then every other week for a maximum of 26 weeks. Part B of the study assessed the long-term safety of Enjaymo and the duration of response to treatment over more than two years of follow-up in patients with FAD. In Part A of the CARDINAL study, the efficacy of Enjaymo was assessed based on the primary composite endpoint (Hb≥12 g/dl or an increase of at least 2 g/dl; no blood transfusion or prohibited drugs between week 5 and week 26) and various secondary endpoints, including improvement in hemoglobin levels, normalization of bilirubin levels and the fatigue score measured on the FACIT-Fatigue scale. Adverse reactions observed in 10% or more of patients were: respiratory tract infections, viral infections, diarrhoea, dyspepsia, cough, arthralgia, arthritis and edema peripheral. Serious adverse reactions were reported in 13% (3/24) of patients treated with Enjaymo. These adverse reactions were: streptococcal sepsis and staphylococcal wound infection (n=1), arthralgia (n=1), and respiratory tract infection (n=1).

About Enjaymo



Enjaymo is a humanized monoclonal antibody designed to selectively target and inhibit the C1 fraction of the classical complement pathway, a component of the innate immune system. By inhibiting the C1 moiety, Enjaymo prevents activation of the complement cascade of the immune system and inhibits hemolysis activated by the C1 complex characteristic of MAF, preventing the abnormal destruction of healthy red blood cells. Enjaymo does not inhibit the lectin pathway or the alternate pathway. Approved by the U.S. Food and Drug Administration (FDA) in February 2022, Enjaymo is the first and only drug indicated to reduce the need for blood transfusions and treat hemolysis, or destruction of red blood cells, in adults with cold agglutinin disease (CAD). The Japanese Ministry of Health, Labor and Welfare approved Enjaymo in June 2022. The European Medicines Agency (EMA) also decided to maintain the orphan drug designation.

About Cold Agglutinin Disease

Cold agglutinin disease is a rare chronic autoimmune hemolytic anemia that causes the immune system to mistakenly attack and destroy red blood cells (hemolysis). Cold Agglutinin Disease is believed to affect 12,000 people in the United States, Europe and Japan. It causes profound fatigue and increases the risk of thromboembolic events and mortality.

About Sanofi

We are an innovative, global health company driven by one purpose: to pursue the miracles of science to improve people’s lives. Our teams, present in a hundred countries, are working to transform the practice of medicine to make the impossible possible. We provide therapeutic solutions that can change the lives of patients and vaccines that protect millions of people around the world, guided by the ambition of sustainable development and our social responsibility.

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

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