EC Approves Enjaymo as 1st Treatment for Anemia in CAD Adults

Decision in Europe comes 9 months after US approval

Steve Bryson, PhD avatar

by Steve Bryson, PhD |

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Enjaymo (sutimlimab-jome) has been approved by the European Commission (EC) to treat anemia caused by the loss of red blood cells — called hemolytic anemia — in adults with cold agglutinin disease (CAD).

The EC’s decision to approve the therapy, the first and only approved therapeutic option for patients with the rare autoimmune disorder, comes on the heels of a recent approval recommendation by the European Medicines Agency.

“This approval highlights our ambition to develop first- and best-in-class medicines that transform people’s lives,” Dietmar Berger, MD, PhD, chief medical officer and global head of development at Sanofi, said in a press release. Sanofi is the company developing and marketing Enjaymo.

“The approval of Enjaymo by the European Commission provides patients, for the first time, with access to a therapy that can make a meaningful difference in the treatment and daily experience of living with CAD,” Berger said.

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1st treatment approved for CAD in Europe

In CAD, the immune system mistakenly attacks and destroys red blood cells, which carry oxygen through the bloodstream, at low temperatures. A loss of red blood cells leads to anemia, which deprives tissues of oxygen.

Symptoms, such as fatigue and pain, usually can be sufficiently controlled by avoiding exposure to cold temperatures. Still, in more severe cases, patients may require medications not approved for CAD — and thus used off-label — and blood transfusions.

“Up until now, patients in Europe had to rely on a combination of cold avoidance, blood transfusions, and off-label treatments to manage their disease,” Berger said.

Enjaymo is designed to block the activation of a part of the immune system directly involved in this attack, called the complement cascade, thereby preventing the destruction of red blood cells. The therapy was approved in the U.S. earlier this year to reduce the need for red blood cell transfusions in CAD adults.

The EU approval was supported by data from two Phase 3 clinical trials: CARDINAL (NCT03347396) and CADENZA (NCT03347422).

In CARDINAL, 24 adults with CAD who had recently been given a blood transfusion received Enjaymo by infusion directly into the bloodstream (intravenously). Participants were given infusions once a week for the first two weeks and then every other week for a total of 26 weeks, or six months. Enjaymo treatment reduced hemolysis and anemia and improved quality of life in these patients.

CADENZA enrolled 42 CAD patients who had not received a blood transfusion in the six months before the study. Using the same dosing regimen as CARDINAL, patients were randomly assigned to Enjaymo or a placebo. Enjaymo was found to lessen anemia, and also fatigue, as well as the need for other CAD therapies.

“Coupled with diagnostic journeys that can last years, the impact of fatigue on quality of life in CAD is often debilitating and is comparable to conditions such as cancer-related anemia and other autoimmune disorders,” said Alexander Röth, MD, from the University of Duisburg-Essen, in Germany.

“Clinicians now have a much-needed therapeutic option to offer to their patients,” he said.