The first condensate-collecting disease (CAD) hemolytic therapy drug! Sanofi supplement C1s inhibitor sutimlimab was rejected by the FDA!

November 18, 2020 // -- Sanofi recently announced that the U.S. Food and Drug Administration (FDA) has issued a full response letter (CRL) to Suttimlimab's Biological Products Licensing Application (BLA), a monoclonal antibody used to treat hemolysis in adult patients with primary coagulation disease (CAD).
sutimlimab targets the underlying causes of CAD hemolysis by selectively inhibiting supplement C1s.
in crL, the FDA noted that some defects were found during pre-approval pre-inspections of third-party facilities responsible for manufacturing (Pre-license Inspection, PLI).
clinical or safety deficiencies associated with the application were found in the CRL.
third-party manufacturers are required to resolve the defects mentioned in the CRL se as satisfactory before the BLA can be approved.
Sanofi will be in close contact with the FDA and third-party manufacturers to reach a solution in a timely manner.
May, the FDA accepted Sutmimab's BLA and granted priority review.
if approved, sutimlimab would be the first and only drug to treat CAD hemolysis.
, the FDA has granted Suttimlimab Breakthrough Drug Qualification (BTD) and Orphan Drug Qualification (ODD).
CAD is a rare, severe, chronic, autoimmune hemolytic anemia in which the complement system in the immune system mistakenly attacks healthy red blood cells and causes them to rupture (hemolytic).
with CAD may experience chronic anemia, severe fatigue, acute hemolytic risk and other potential complications, including an increased risk of thrombosis events and early death.
estimated that there are about 5,000 cases of CAD in the United States.
the submission of the sutimlimab BLA, based on the results of an open-label single-arm critical PHASE III CARDINAL study conducted in primary CAD patients.
data, published at the 61st Annual Meeting of the American Society of Hematology (ASH2019) in 2019, confirm that sutimlimab meets the main compound efficacy indicator, defined as the proportion of patients who meet all of the following composite criteria: at the point in time for treatment evaluation (23 week, 25 weeks, 26 weeks average) Hemoglobin levels increase relative to the baseline ≥2g/dL or reach hemoglobin levels ≥12g/dL, no blood transfusions during weeks 5-26.
In addition, trials have shown that sutimlimab has reached a secondary end point: showing improvements in key indicators of the disease process, including improvements in hemoglobin, normalization of bilium, and improvements in the evaluation of chronic disease treatment function assessment (FACIT) fatigue scores.
sutimlimab is a potentially pioneering, research-based, humanized monoclonal antibody specifically designed to selectively target and suppress serine protease C1s in the C1 complex, which is the first step in activating the immune system's classic complement pathway.
classical complement pathway is part of the innate immune system, and its activation is the central mechanism of CAD hemolysis.
by targeting the suppression of C1s, sutimlimab is believed to block the activation of the classical complement pathway, thereby preventing the activation of C1 hemolysis in CAD.
sutimlimab has novel mechanism of effect and high target specificity, which can selectively inhibit the upstream of classical complement pathways in the course of disease, while retaining the complete alternative complement pathway and coagulation supplement pathway and its immunosuppression function.
, Sanofi is currently evaluating Sutimlimab in patients with CAD who have not recently had blood transfusions in phase III CADENZA trials, and the company is also investigating Sutimlimab's use in the treatment of immunoplate reduced cyanosis (ITP) patients.
() Original origin: FDA Issues Complete Response Letter for sutimlimab, an investigational treatment for hemolysis in adults with cold agglutinin disease