Sanofi’s Enjaymo (sutimlimab) has been approved by the European Commission (EC) for the treatment of haemolytic anaemia in adult patients with cold agglutinin disease (CAD), the company announced.
Enjaymo is currently the only approved treatment for CAD, a rare type of autoimmune haemolytic anaemia where part of the body’s immune system mistakenly destroys healthy red blood cells.
The disease affects 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.
“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 anaemia and other autoimmune disorders,” said Professor Alexander Röth, Department of Hematology and Stem Cell Transplantation, University Hospital, University of Duisburg-Essen, Germany.
The company’s EC submission included data from two phase 3 trials: CADENZA, in patients without a recent history of blood transfusion, and CARDINAL, in patients who have had a recent blood transfusion.
In the CADENZA Part A trial, Enjaymo met its primary composite endpoint and all secondary endpoints, demonstrating inhibition of haemolysis, increase in haemoglobin levels, and clinically meaningful improvement in The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores.
The company also reported that Enjaymo demonstrated an acceptable safety profile and was generally well-tolerated.
In the CARDINAL Part A trial, the efficacy of Enjaymo was assessed based on the achievement of a primary composite endpoint and different secondary endpoints, including improvements in haemoglobin, normalisation of bilirubin, and FACIT-fatigue score.
Serious adverse reactions were reported in 13% of patients who received Enjaymo. These were streptococcal sepsis and staphylococcal wound infection, arthralgia and respiratory tract infection.
Enjaymo was approved by the US 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 haemolysis in adults with CAD.
Commenting on the latest regulatory approval, Dietmar Berger, chief medical officer, global head of development at Sanofi, said: “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.
“The approval of Enjaymo by the EC 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.”