Sanofi’s treatment, Enjaymo, for hemolytic anemia in adult patients with cold agglutinin disease (CAD), has been granted marketing authorization by the European Commission (EC).
Enjaymo is the first and only approved therapeutic option for the rare, serious and chronic autoimmune disease where the body mistakenly attacks healthy red blood cells and causes their rupture, known as hemolysis.
Dietmar Berger, global head of development and chief medical officer at Sanofi, 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.
Enjaymo – only approved treatment
“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.”
Enjaymo is currently the only approved treatment for CAD and is a first-in-class humanized monoclonal antibody that is designed to selectively target and inhibit the classical complement pathway specific serine protease, C1s. It will be available as a 50mg/mL solution for infusion.
Alexander Röth, Department of Hematology, University Hospital, Duisburg-Essen, Germany, said: “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. Clinicians now have a much-needed therapeutic option to offer to their patients.”
Durability of response to Enjaymo
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 (6.5g or 7.5g) 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.
Improvements in hemoglobin
Enjaymo demonstrated an acceptable safety profile and was generally well-tolerated. 96% of patients in the Enjaymo group and 100% of patients in the placebo group experienced at least one treatment emergent adverse event (TEAE). Headache (22.7% vs 10.0%), hypertension (22.7% vs 0%), rhinitis (18.2% vs 0%), Raynaud phenomenon (18.2% vs 0%), and acrocyanosis (13.6% vs 0%) were reported more frequently in Enajymo-treated patients compared with placebo.
In the CARDINAL Part A trial, patients received a fixed weight-based dose (6.5g or 7.5g) of Enjaymo via intravenous infusion on Day 0, Day 7, and then once every other week up to Week 26. Part B of the study evaluated the long-term safety as well as durability of response to Enjaymo in patients with CAD over a 2-year follow up. In the CARDINAL Part A study, the efficacy of Enjaymo was assessed based on the achievement of a primary composite endpoint and different secondary endpoints, including improvements in hemoglobin, normalization of bilirubin, and FACIT-fatigue score. The most common adverse reactions occurring in 10% or more of patients were respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral edema.
Serious adverse reactions were reported in 13% (3/24) of patients who received Enjaymo. These serious adverse reactions were streptococcal sepsis and staphylococcal wound infection, arthralgia, and respiratory tract infection.