FDA Expands Label of Sutimlimab-jome for Rare Disease to Include Adults Without Transfusion History

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The monoclonal antibody serves as the first and only therapy for treating hemolysis in adults with cold agglutinin disease, an extremely rare condition.

The FDA approved Sanofi's supplemental Biologics License Application (sBLA) for sutimlimab-jome (Enjaymo). The label will now include adults without a history of transfusion who have cold agglutinin disease (CAD). The treatment was first granted FDA approval last February for adults with the rare blood disorder that had received prior transfusions.

Cold agglutinin disease is an extremely rare, life-threatening condition that's diagnosed in one of every million people in the US. Red blood cells are incorrectly attacked by the patient's immune system resulting in hemolysis, or the rupturing of otherwise healthy red blood cells.

Sutimlimab-jome is a first-in-class monoclonal antibody and the first and only therapy for hemolysis in adults with cold agglutinin disease. It works by selectively targeting and inhibiting the classical complement pathway-specific serine protease, C1s.

Data from the double-blinded, placebo-controlled CADENZA clinical trial supported the FDA's latest decision. Long-term benefits and safety of the treatment was evaluated in patients without a recent history of transfusion.

Investigators observed patients treated with sutimlimab-jome showed inhibited hemolysis and reduced need for transfusions and disease-related treatments, therefore meeting the composite primary endpoint. Additionally, data regarding the secondary endpoint exhibited increased hemoglobin levels, as well as decreased billirubin levels and lactate dehydrogenase (LDH) levels.

“This milestone underscores our ongoing commitment to improve outcomes for the rare blood disorder community," Bill Sibold, Executive Vice President, Head of Specialty Care, Sanofi, said in a statement. "As a first-in-class therapy, Enjaymo can help people living with cold agglutinin disease better manage their symptoms, including the impact of fatigue, which may positively affect their daily life.”