FDA Approves Sutimlimab for Cold Agglutinin Disease

The FDA last week approved sutimlimab for use in patients with a rare type of anemia. The biologic decreases the need for red blood cell transfusion due to hemolysis in adults with cold agglutinin disease (CAD), a rare autoimmune hemolytic anemia activated by the classic complement pathway.

Sutimlimab electively targets the C1s protein, a C1 complex serine protease which activates the pathway.

The drug will be marketed by Sanofi under the name Enjaymo and it is 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.

The 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 a 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.

The therapy previously received FDA Breakthrough Therapy and Orphan Drug designation, as well as Priority Review. Outside of the United States, it has been submitted to regulatory authorities in Europe and Japan.

Sutimlimab is expected to be available in the United States in the coming weeks; Sanofi said the US list price, or wholesale acquisition cost will be $1800 per vial.