FDA Grants Sanofi Priority Review for Experimental CAD Treatment

Rare Daily Staff

The U.S. Food and Drug Administration (FDA) has accepted Sanofi’s biologics license application for its experimental monoclonal antibody sutimlimab for adults with cold agglutinin disease and granted it Priority Review.

The marketing application is the first to be accepted for the treatment of patients with cold agglutinin disease.

Priority review is granted to drugs that treat a serious condition and if approved would provide a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The FDA is expected to act on the application by November 13, 2020.

Cold agglutinin disease (CAD) is a chronic autoimmune hemolytic anemia that causes the body’s immune system to mistakenly attack healthy red blood cells and cause their rupture (hemolysis). CAD patients may experience chronic anemia, profound fatigue, acute hemolytic crisis, and other potential complications, including an increased risk of thromboembolic events and early death. An estimated 5,000 people live with CAD in the United States.

Sutimlimab, an experimental monoclonal antibody, targets the underlying cause of hemolysis in CAD by selectively targeting and inhibiting C1s in the classical complement pathway, which is part of the innate immune system. By blocking C1s, it is thought that sutimlimab halts C1-activated hemolysis in CAD. The inhibition of the classical pathway at C1s aims to retain immune surveillance functional activities of the alternative or lectin complement pathways.

“People living with cold agglutinin disease currently have no approved treatment option and experience chronic anemia and profound fatigue, which have a persistent and serious impact on their lives,” said John Reed, global head of research and development at Sanofi.

The BLA submission is based on results from part A of the open label, single arm pivotal phase 3 CARDINAL study in 24 patients with primary CAD. The data, which were presented at the 61st annual meeting of the American Society of Hematology, demonstrated that sutimlimab had a clinically meaningful effect on complement-mediated hemolysis, the cause of anemia and fatigue. The trial showed sutimlimab also met its secondary endpoints by indicating improvements in disease process, including improvements in hemoglobin, normalization of bilirubin, and improvements in Functional Assessment of Chronic Illness Therapy-Fatigue Score.

Sanofi is evaluating sutimlimab in the on-going phase 3 CADENZA trial for CAD patients who have not recently had a blood transfusion and separately, investigating sutimlimab for patients with immune thrombocytopenic purpura. Sutimlimab has been granted Breakthrough Therapy and Orphan Drug designation by the FDA.

Photo: John Reed, global head of research and development at Sanofi