The US Food and Drug Administration (FDA) has approved sutimlimab (Enjaymo™) as the first treatment designated for cold agglutinin disease (CAD), drugmaker Sanofi and the agency announced.
Sutimlimab is a humanized monoclonal antibody that selectively inhibits C1s in the classical complement pathway, thereby inhibiting the activation of the complement cascade without inhibiting the lectin and alternative pathways. This way, it decreases the need for red blood cell transfusion due to hemolysis in patients with CAD.
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“Until now, people living with cold agglutinin disease haven’t had an approved treatment option to manage the constant destruction of red blood cells,” said Bill Sibold, executive vice president and head of specialty care at Sanofi. “Without healthy, viable red blood cells, a chain reaction of debilitating signs and symptoms can be triggered, starting with severe anemia. Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start.”
The approval is based on the results of an open-label, single-arm, pivotal phase 3 clinical trial called CARDINAL that assessed the safety and efficacy of sutimlimab in 24 patients with CAD who had a recent blood transfusion.
The primary endpoints of the trial were the number of participants with treatment-emergent and serious adverse events and the proportion of patients showing a response. A response was defined as an increase in hemoglobin levels, an indirect measurement of red blood cells that were not destroyed, to 12 g/dL or more, or an increase in hemoglobin levels of 2 g/dL or more from baseline at 23, 25, and 26 weeks and no blood transfusion or medications prohibited by the trial protocol from weeks 5 to 26.
The secondary endpoints included the mean change from baseline in bilirubin, lactate dehydrogenase, and hemoglobin levels, quality of life, and the number of blood transfusions and blood units transfused after the first 5 weeks.
More than half (54%) of the participants responded to the treatment. The most common adverse reactions were respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral edema. Serious adverse reactions were wound infection, arthralgia, and respiratory tract infection.
According to Sanofi, the treatment will be available in the U.S. in the coming weeks.
The New Drug Application for sutimlimab has previously granted orphan drug designation, breakthrough therapy designation, and priority review by the FDA.
FDA approves Enjaymo™ (sutimlimab-jome), first treatment for use in patients with cold agglutinin disease. News release. Sanofi; February 4, 2022.
A study to assess the efficacy and safety of BIVV009 (sutimlimab) in participants with primary cold agglutinin disease who have a recent history of blood transfusion (cardinal study). US National Library of Medicine. Last updated October 5, 2021. Accessed February 8, 2022.