FDA grants priority review of sutimlimab, potential first approved treatment of hemolysis for cold agglutinin disease.- Sanofi

The FDA has granted priority review of Sanofi’s Biologics License Application (BLA) for sutimlimab for the treatment of hemolysis in adult patients with cold agglutinin disease (CAD). Sutimlimab, an investigational monoclonal antibody, targets the underlying cause of hemolysis in CAD by selectively inhibiting complement C1s. If approved, sutimlimab would be the first and only approved treatment for these patients. The target action date for the FDA decision is November 13, 2020.

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 in the U.S. live with CAD.

The BLA submission is based on results from part A (n=24) of the open label, single arm pivotal Phase III CARDINAL study in patients with primary CAD. The data were presented in the Late-Breaking Abstracts Session at the 61st Annual Meeting of the American Society of Hematology and demonstrated sutimlimab met its primary composite efficacy endpoint defined as the proportion of patients who demonstrated an increase from baseline in Hgb level greater than 2 g/dL or normalization of Hgb level greater than 12 g/dL at the treatment assessment time point (mean value from weeks 23, 25, and 26) and no blood transfusion from week 5 through week 26. 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 III 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..