Enjaymo Recommended for EU Approval to Treat Adults With CAD

If approved, it would be the first therapy in the EU for hemolytic anemia in CAD

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A committee of the European Medicines Agency has recommended Enjaymo (sutimlimab-jome) be approved for hemolytic anemia, or anemia due to the destruction of red blood cells, in adults with cold agglutinin disease (CAD).

The positive opinion from the agency’s Committee for Medicinal Products for Human Use now will be reviewed by the European Commission, which is expected to make its final decision before the end of the year, according to a press release from Sanofi, the company developing Enjaymo.

In CAD, the body’s immune system mistakenly attacks and destroys healthy red blood cells — those that carry oxygen through the bloodstream — leading to anemia and depriving tissues of oxygen.

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Enjaymo is designed to block the activation of the complement cascade, a part of the immune system that plays a key role in this attack. It acts by specifically targeting and blocking a complement protein called C1, thereby preventing the destruction of red blood cells that’s driven by the excessive activity of the complement cascade.

The U.S. approved the treatment earlier this year for adults with CAD to reduce the need for red blood cell transfusions due to hemolysis, making it the first therapy approved for that indication in the country. If approved by the European Commission, it would become the first approved CAD treatment in Europe as well.

The European committee’s positive opinion was supported by findings from two Phase 3 clinical trials: one called CARDINAL and another called CADENZA.

The CARDINAL trial (NCT03347396) enrolled 24 people with CAD who had recently received a blood transfusion. All participants were treated with a fixed weight-based dose (6.5 or 7.5 g) of Enjaymo, administered by infusion directly into the bloodstream. Infusions were given weekly for the first two weeks, and then every other week for a total of 26 weeks.

Trial results showed Enjaymo treatment eased anemia, reduced hemolysis, and improved quality of life.

CADENZA (NCT03347422) enrolled 42 CAD patients who had not recently received a blood transfusion (in the six months preceding study entry). Participants were randomly assigned to receive Enjaymo or a placebo, given via the same dosing schedule as that used in CARDINAL.

Study results showed Enjaymo eased anemia and fatigue and also reduced the need for other CAD treatments.