FDA Grants Priority Review to Sutimlimab, a Potential First-in-Class Treatment for Hemolysis in Adults with Cold Agglutinin Disease

May 15, 2020 News /BioValleyBIOON/ -- Sanofi recently announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for sutimlimab for the treatment of hemolysis in adult patients with primary cold agglutinin disease (CAD) and granted it priority review. Sutimlimab is an investigational monoclonal antibody that targets the underlying cause of hemolysis in CAD by selectively inhibiting complement C1s.

If approved, sutimlimab will become the first and only drug for treating hemolysis in CAD.FDAThe target action date for the BLA has been set as November 13, 2020.

CAD is a rare, severe, chronic,AutoimmunityHemolyticAnemiaIn this disease, the complement system of the immune system mistakenly attacks healthy red blood cells in the body, causing them to rupture (hemolysis). Patients with CAD may experience chronic anemia, severe fatigue, acute hemolytic crises, and other potential complications, including an increased risk of thromboembolic events and premature death. It is estimated that there are approximately 5,000 patients with CAD in the United States.

The submission of the Biologics License Application (BLA) for sutimlimab was based on results from Part A (n=24) of the pivotal, open-label, single-arm Phase III CARDINAL study conducted in patients with primary cold agglutinin disease (CAD). These data were presented at the 61st American Society of Hematology Annual Meeting (ASH 2019) and confirmed that sutimlimab met the primary composite efficacy endpoint, defined as the proportion of patients achieving all of the following criteria: an increase in hemoglobin level of ≥2 g/dL from baseline or attainment of a hemoglobin level ≥12 g/dL (based on the mean values at weeks 23, 25, and 26), and no transfusions during weeks 5–26. Furthermore, the trial demonstrated that sutimlimab also met secondary endpoints, showing improvements in key indicators of disease progression, including increased hemoglobin levels, normalization of bilirubin, and improved scores on the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale.

Dr. John Reed, Global Head of Research and Development at Sanofi, stated, “Currently, there are no approved treatment options for patients with cold agglutinin disease, who experience chronic anemia and severe fatigue, causing persistent and significant impacts on their lives. Results from the 26-week pivotal Phase III CARDINAL study clearly demonstrate that sutimlimab has a clinically meaningful therapeutic effect on complement-mediated hemolysis (the cause of anemia and fatigue). If approved, sutimlimab will be the first and onlyFDAapproved treatment regimen that uniquely addresses C1-mediated hemolysis and helps alleviate the chronic disease burden in patients with CAD.”

Sutimlimab is a potential first-in-class, investigational, humanized monoclonal antibody specifically designed to selectively target and inhibit C1s, the serine protease within the C1 complex, which constitutes the initial step in activating the classical complement pathway of the immune system. The classical complement pathway, a component of the innate immune system, plays a central role in the hemolysis associated with cold agglutinin disease (CAD). By selectively inhibiting C1s, sutimlimab is believed to block the activation of the classical complement pathway, thereby preventing C1-mediated hemolysis in CAD.

Sutimlimab features a novel mechanism of action and high target specificity, selectively inhibiting the upstream classical complement pathway during disease progression while preserving the intact alternative and lectin complement pathways and their immune surveillance functions.

Currently, Sanofi is evaluating sutimlimab in patients with cold agglutinin disease (CAD) who have not received recent transfusions in the Phase III CADENZA trial, and the company is also investigating sutimlimab for the treatment of patients with immune thrombocytopenia (ITP).

Previously, the United StatesFDASutimlimab has been granted Breakthrough Therapy Designation (BTD) and Orphan Drug Designation (ODD). Sutimlimab is an investigational drug currently in clinical development, and its safety and efficacy have not yet been evaluated by any regulatory authority. (Bioon.com)

Original Source:FDA grants priority review of sutimlimab, potential first approved treatment of hemolysis in adult patients with Cold Agglutinin Disease