Priority review to sutimlimab for treating hemolysisJune 10, 2020
The US 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, Sanofi said.
The BLA submission is based on results from part A (n=24) of the open-label, single-arm pivotal phase 3 CARDINAL study in patients with primary CAD. Sutimlimab met its primary composite efficacy endpoint defined as the proportion of patients who demonstrated an increase from baseline in Hgb level =2 g/dL or normalisation of Hgb level =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 the disease process, including improvements in hemoglobin, normalisation of bilirubin, and improvements in Functional Assessment of Chronic Illness Therapy-Fatigue Score.
Sutimlimab is designed to selectively target and inhibit 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.
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.