FDA Approves Sutimlimab as First Drug for Cold Agglutinin Disease

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The monoclonal antibody is designed to reduce the need for red blood cell transfusions to hemolysis among adults with the rare disease.

The US Food and Drug Administration (FDA) has approved sutimlimab-jome (ENJAYMO) for the treatment of adults with cold agglutinin disease (CAD).

The approval, granted to Sanofi, indicates the intravenous (IV) injection complement inhibitor monoclonal antibody for the decreased need for red blood cell transfusions to hemolysis among adults with the rare disease.

Sutimlimab is now the first treatment approved for patients with CAD, which affects approximately 16 per 1 million people globally, and develops in approximately 1 per 1 million people annually.

Sanofi’s approval was supported by data from the 26-week, open-label, single-arm, phase 3 CARDINAL study, which studied sutimlimab in 24 patients with CAD who had recent history of blood transfusions.

In the trial, the investigative drug met the primary efficacy composite endpoint of proportion of patients to achieve normalized hemoglobin level ≥12 g/dL or a demonstrated increase from baseline in hemoglobin level ≥2 g/dL at the treatment assessment time point, as well as no reported blood transfusions nor prohibited medications from weeks 5 through 26.

The therapy additionally achieved secondary endpoint improvements in mean increased hemoglobin levels at week 3 (2.29 g/dL) and week 26 (3.18 g/dL) versus baseline, as well as in mean reduction of bilirubin levels by 2.23 mg/dL (95% CI, -2.49 to -1.98) from baseline 3.23 mg/dL.

Common adverse events observed in patients from the CARDINAL trial (≥10% of patients) included respiratory tract infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral edema.

Based on clinical assessment, sutimlimab is recommended at doses based on patient body weight (6500 mg for 39-75 kg; 7500 mg for >75 kg), administered intravenously weekly for 2 weeks then every 2 weeks thereafter.

In a statement accompanying the approval, Catherine Broome, MD, associate professor of Medicine at Georgetown University Lombardi Comprehensive Cancer Center and CARDINAL principal investigator, emphasized the burden patients with CAD faced without prior resolution delivered by an indicated therapy.

“For people living with cold agglutinin disease, it is as if their body’s immune system is waging a war on itself,” Broome said. “The relentless destruction of healthy red blood cells is a daily, silent reality for people with CAD. For the first time, we have a treatment that targets complement-mediated hemolysis, which is the underlying cause of the red blood cell destruction in many CAD patients.”