Enjaymo receives EU marketing authorization for hemolytic anemia

Sanofi’s product, Enjaymo, for the treatment of hemolytic anemia in adult patients with cold agglutinin disease (CHD) has received marketing authorization from the European Commission (EC).

Enjaymo is the first and only approved therapeutic option for rare, serious and chronic diseases autoimmune diseases where the body mistakenly attacks healthy red blood cells and causes them to break, known as hemolysis.

Dietmar Berger, Global Head of Development and Chief Medical Officer at Sanofi, said: “It approval underlines our ambition to develop first and best-in-class medicines that change people’s lives. Until now, patients in Europe had to rely on a combination of avoiding colds, blood transfusions and off-label treatments to combat the disease.

Enjaymo is an approved treatment only

“Approval of Enjaymo by European Commission for the first time, gives patients access to a therapy that can make a big difference in the treatment and daily experience of living with CHD.”

Enjaymo is currently the only approved treatment for CHD and the first in its class to be humanized monoclonal antibodies which is designed to selectively target and inhibit the classical complement pathway, the specific serine protease, C1s. It will be available as a 50 mg/ml solution for infusion.

Alexander Reth, Department of Hematology, University Hospital Duisburg-Essen, Germany, said: “Combined with diagnostic visits that can last for years, the impact of fatigue on the quality of life in CHD is often debilitating and comparable to diseases such as cancer-related anemia and other autoimmune diseases. Clinicians now have a much-needed therapeutic option to offer their patients.”

Durability of response to Enjaymo

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

In the CADENZA Part A study, eligible patients were randomized 1:1 to receive a fixed weight-based dose (6.5 g or 7.5 g) of Enjaymo or placebo via intravenous infusion on day 0, day 7, and once every other week. until the 26th week.

An open-label Part B study evaluated the long-term safety as well as the duration of response to Enjaymo in patients with CHD. In the CADENZA Part A study, Enjaymo met its primary composite endpoint and all secondary endpoints and demonstrated inhibition of hemolysis, increased hemoglobin, and clinically significant improvement in Functional Assessment of Chronic Disease Therapy (FACIT) – fatigue.

Improvement of 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’s phenomenon (18.2% vs. 0%), and acrocyanosis ( 13.6% vs. 0%) were reported more frequently in patients treated with Enajymo compared to placebo.

In the CARDINAL Part A study, patients received a fixed body weight-based dose (6.5 g or 7.5 g) of Enjaymo by intravenous infusion on day 0, day 7, and then every other week until week 26. Part B of the study evaluated the long-term safety as well as the duration of response to Enjaymo in patients with CAD during a 2-year follow-up. In the CARDINAL Part A study, the efficacy of Enjaymo was evaluated based on the achievement of the primary composite endpoint and various secondary endpoints, including improvement in hemoglobin, normalization of bilirubin, and the 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 receiving Enjaymo. These serious adverse reactions included streptococcal sepsis and staphylococcal wound infection, arthralgia, and respiratory tract infection.