The EC approval for Enjaymo was driven by the findings from the CADENZA and CARDINAL phase 3 clinical trials
Sanofi has received marketing authorisation from the European Commission (EC) for Enjaymo (sutimlimab) for the treatment of haemolytic anaemia in adults having cold agglutinin disease (CAD).
To be available as a 50mg/mL solution for infusion, Enjaymo is presently the only approved treatment for the rare, serious, and chronic autoimmune hemolytic anemia, said the French pharma company.
According to Sanofi, the humanised monoclonal antibody has been developed to preferentially target and inhibit C1s, which is a classical complement pathway-specific serine protease.
Sanofi chief medical officer and development global head Dietmar Berger said: “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.
“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.”
The EC approval for Enjaymo was based on the findings from the CADENZA and CARDINAL phase 3 clinical trials.
In the CADENZA Part A study, the humanised monoclonal antibody met its primary composite endpoint as well as all secondary endpoints. It also showed inhibition of haemolysis, increased haemoglobin levels, and enhanced The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores.
The pharmaceutical company said that in the CARDINAL Part A study, the efficacy of Enjaymo was evaluated on the basis of achievement of the primary composite endpoint and different secondary endpoints, including improvements in haemoglobin, FACIT-fatigue score, and normalisation of bilirubin.
CADENZA was a double-blind, placebo-controlled study of patients with cold agglutinin disease who did not have a blood transfusion in the last six months. CARDINAL was a 26-week open-label, single-arm trial in patients with the same disease who completed a recent blood transfusion.