FDA Approves Enjaymo for Treatment of Cold Agglutinin Disease

The FDA has approved sutimlimab-jome (Enjaymo, Sanofi) to decrease the need for red blood cell transfusion because of hemolysis in adults with cold agglutinin disease (CAD).

Enjaymo is the first and only approved treatment for individuals with CAD and works by inhibiting the destruction of red blood cells.

“Until now, people living with cold agglutinin disease haven’t had an approved treatment option to manage the constant destruction of red blood cells. Without healthy, viable red blood cells, a chain reaction of debilitating signs and symptoms can be triggered, starting with severe anemia,” Bill Sibold, executive vice president and head of specialty care at Sanofi, said in a statement

“Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start,” he said.

CAD, a rare autoimmune hemolytic anemia, is caused by antibodies called cold agglutinins binding to the surface of red blood cells, which starts a process that causes the body’s immune system to mistakenly attack healthy red blood cells and cause their rupture.

CAD affects an estimated 5000 individuals in the United States, according to the statement.

Enjaymo is a humanized monoclonal antibody that is designed to selectively target and inhibit C1s in the classical complement pathway, which is part of the innate immune system. Emjaymo does not inhibit the lectin and alternative pathways.

The approval of Enjaymo in the United States is based on positive results from the 26-week open-label, single-arm pivotal phase 3 CARDINAL study for individuals with CAD who have a recent history of blood transfusion.

In the study, Enjaymo met its primary efficacy endpoint, whichwas a composite endpoint defined as the proportion of individuals who achieved normalization of hemoglobulin (Hgb) level greater than or equal to 12 g/dL or demonstrated an increase from baseline in Hgb level greater than or equal to 2 g/dL at the treatment assessment time point. Additionally, there had to be no blood transfusions from week 5 through 26 or medication prohibited per the protocol from weeks 5 through 26.

About 54% of individuals met the composite primary endpoint, with 63% of individuals achieving a hemoglobin greater or equal to 12 g/dL or an increase of at least 2 g/dL, while 71% of individuals remained transfusion-free after week 5 and 92% of individuals did not use other CAD-related treatments.

The secondary endpoints were also met, including improvements in hemoglobin and normalization of bilirubin.

Individuals enrolled experienced a mean increase in hemoglobin levels of 2.29 g/dL at week 3 and 3.18 g/dL at the 26-week treatment assessment timepoint from the mean baseline level of 8.6 g/dL.

The mean reduction in bilirubin levels was by negative 2.23 mg/dL from a mean baseline level of 3.23 mg/dL.

The most common adverse events (AEs) were arthralgia, arthritis, cough, diarrhea, dyspepsia, peripheral edema, and respiratory tract or viral infection.. Serious AEs were reported in about 13% of individuals who received Enjaymo and included arthralgia, respiratory tract and staphylococcal wound infection, and streptococcal sepsis.

Following the completion of the study period, eligible individuals continued to receive Enjaymo in an extension study.

Reference

FDA approves Enjaymo (sutimlimab-jome), first treatment for use in patients with cold agglutinin disease. Globe Newswire. News release. February 4, 2022. Accessed February 8, 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