The Food and Drug Administration (FDA) has approved Enjaymo™ (sutimlimab-jome) to decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease (CAD).
Sutimlimab is an immunoglobulin G subclass 4 (IgG4) monoclonal antibody that inhibits the classical complement pathway and specifically binds to complement protein component 1. This inhibition prevents deposition of complement opsonins on the surface of RBCs, resulting in inhibition of hemolysis in patients with CAD.
The approval was based on data from the open-label, single-arm phase 3 CARDINAL study (ClinicalTrials Identifier: NCT03347396), which evaluated the efficacy and safety of sutimlimab in 24 patients with primary CAD who had a recent blood transfusion. Patients received an intravenous infusion of sutimlimab through week 26.
The primary end point was the proportion of patients with a response, defined by an increase in hemoglobin (Hgb) of at least 2g/dL from baseline or reaching a Hgb level of at least 12g/dL at the 26-week treatment assessment timepoint, as well as the absence of blood transfusions from weeks 5 to 26 or any other CAD-related treatments.
Results showed that 54% (n=13) of patients achieved the composite endpoint. Sixty-three percent (n=15) of patients had an increase in Hgb of at least 2g/dL or reached an Hgb of at least 12g/dL, while 71% (n=17) did not receive RBC transfusion after week 5 and 92% (n=22) did not use other CAD-related treatments. Treatment with sutimlimab was also associated with improvements in total bilirubin (mean reduction in bilirubin levels [n=14]: -2.23mg/dL [95% CI, -2.49, -1.98]) and lactate dehydrogenase (least squared mean change: -126 [95% CI, -218, -35]). At week 3, the mean increase in Hgb level was observed to be 2.29g/dL (SE: 0.308). At the 26-week treatment assessment timepoint, the mean increase in Hgb was 3.18g/dL (SE: 0.476).
The most common adverse reactions reported with sutimlimab included respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral edema.
Enjaymo is supplied as a 1100mg/22mL (50mg/mL) solution in a single-dose vial. The treatment, which is expected to be available in the coming weeks, is administered via intravenous infusion.
- FDA approves Enjaymo™ (sutimlimab-jome), first treatment for use in patients with cold agglutinin disease. News release. February 4, 2022. https://www.globenewswire.com/news-release/2022/02/04/2379517/0/en/FDA-approves-Enjaymo-sutimlimab-jome-first-treatment-for-use-in-patients-with-cold-agglutinin-disease.html
- Enjaymo. Package insert. Sanofi; 2022. Accessed February 7, 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761164s000lbl.pdf