European Commission approves Sanofi’s Enjaymo for Treatment of Hemolytic Anemia in Adults with Cold Agglutinin Disease

The European Commission has granted marketing authorization for Sanfoi’s Enjaymo for the treatment of hemolytic anemia in adult patients with cold agglutinin disease, a rare, serious, and chronic autoimmune hemolytic anemia where the body’s immune system mistakenly attacks healthy red blood cells and causes their rupture.

Cold agglutinin disease (CAD) is a rare type of autoimmune hemolytic anemia, where part of the body’s immune system mistakenly destroys healthy red blood cells (hemolysis). CAD impacts the lives of an estimated 12,000 people in the US, Europe, and Japan and is associated with profound fatigue and increased risk of thromboembolic events and mortality.

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. By blocking C1s, Enjaymo inhibits the activation of the complement cascade in the immune system and inhibits C1-activated hemolysis in CAD to prevent the abnormal destruction of healthy red blood cells.

Enjaymo was approved by the U.S. Food and Drug Administration in February 2022 as the first and only treatment indicated to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD. The Japanese Ministry of Health, Labor and Welfare approved Enjaymo in June 2022. The European Medicines Agency also made the decision to maintain orphan designation.

The EC approval is based on data from two phase 3 clinical trials: CADENZA, a double-blind, placebo-controlled clinical trial of adults with CAD without a recent history of blood transfusion (within the past 6 months), and CARDINAL, a 26-week open label, single-arm pivotal study in patients with CAD who have had a recent blood transfusion.

In the CADENZA Part A trial, eligible patients were randomized 1:1 to receive a fixed weight-based dose of Enjaymo or placebo via intravenous infusion on Day 0, Day 7, and then once every other week up to Week 26. The open-label Part B of the study assessed long-term safety as well as durability of response to Enjaymo in patients with CAD. In the CADENZA Part A study, Enjaymo met its primary composite endpoint and all secondary endpoints and demonstrated inhibition of hemolysis, increase in hemoglobin levels, and clinically meaningful improvement in The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores.

Enjaymo demonstrated an acceptable safety profile and was generally well-tolerated. Some 96 percent of patients in the Enjaymo group and 100 percent of patients in the placebo group experienced at least one treatment emergent adverse event (TEAE). Headache (22.7 percent vs 10.0 percent), hypertension (22.7 percent vs 0 percent), rhinitis (18.2 percent vs 0 percent), Raynaud phenomenon (18.2 percent vs 0 percent), and acrocyanosis (13.6 percent vs 0 percent) were reported more frequently in Enajymo-treated patients compared with placebo.

“This approval highlights our ambition to develop first- and best-in-class medicines that transform people’s lives. 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,” said Dietmar Berger, chief medical officer and global head of development at Sanofi. “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.”

Dietmar Berger, chief medical officer and global head of development at Sanofi

Author: Rare Daily Staff