Cambridge, MA, January 25, 2023. The US Food and Drug Administration (FDA) has approved the expanded indication for Enjaymo® (sutimlimab-jome), the first and only treatment of hemolysis in adults with cold agglutinin disease (CAD), to include patients with or without a history of transfusions. The efficacy supplemental Biologics License Application was evaluated under priority review and included long-term data from the pivotal CADENZA and CARDINAL studies.
Enjaymo was first FDA-approved in February 2022 based on data from Part A of the CARDINAL study, a single arm, open label study in patients with CAD with a recent blood transfusion (within the past 6 months).
Bill Sibold
Executive Vice President, Head of Specialty Care at Sanofi
“This milestone underscores our ongoing commitment to improve outcomes for the rare blood disorder community. As a first-in-class therapy, Enjaymo can help people living with cold agglutinin disease better manage their symptoms, including the impact of fatigue, which may positively affect their daily life.”
Expanded label supported by findings from CADENZA and CARDINAL
The expansion of the US prescribing information is based primarily on the Phase 3 CADENZA study, a double-blind, placebo-controlled clinical trial of adults with CAD, without a recent history of blood transfusion (within the past 6 months). The trial met its primary composite endpoint, demonstrating inhibition of hemolysis as well as avoidance of transfusions and use of CAD-related treatments. Secondary endpoint data showed an increase in hemoglobin levels, decrease in bilirubin levels, and decrease in lactate dehydrogenase (LDH) levels.
Patients receiving Enjaymo also reported a statistically significant improvement of symptoms and impacts of fatigue as measured by The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue (LS mean difference of 8.93; 95% CI: 4.0-13.85, p <0.001). The overall safety results were consistent with the safety profile of Enjaymo from the CARDINAL study.
Part B of the CARDINAL and CADENZA studies demonstrated the long-term safety as well as durability of response to Enjaymo in patients with CAD over a 2-year follow up or 1-year follow up, respectively. In both CADENZA and CARDINAL studies, signs and symptoms of recurrent hemolysis were observed nine weeks after the last dose of Enjaymo in Part B. The most common adverse reactions in the CADENZA study (Part A) (incidence ≥18%) are rhinitis, headache, hypertension, acrocyanosis, and Raynaud’s phenomenon. The most common adverse reactions in the CARDINAL study (incidence ≥25%) are urinary tract infection, respiratory tract infection, bacterial infection, dizziness, fatigue, peripheral edema, arthralgia, cough, hypertension, and nausea. Under the expanded label, Enjaymo is now also available in an undiluted preparation, which can decrease the bi-weekly infusion time to one hour.
A breakthrough treatment proven to help address impacts of CAD
Enjaymo is currently the only approved treatment of hemolysis for CAD. It is a first-in-class humanized monoclonal antibody that is designed to selectively target and inhibit the classical complement pathway specific serine protease, C1s. By blocking C1s of the classical complement pathway, Enjaymo inhibits C1-activated hemolysis in CAD to prevent hemolysis, while leaving the lectin and alternative pathways intact.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR US PATIENTS
INDICATION
ENJAYMO® is a prescription medicine used to treat the breakdown of red blood cells (hemolysis) in adults with cold agglutinin disease (CAD). It is not known if ENJAYMO is safe and effective in children.
IMPORTANT SAFETY INFORMATION
Do not receive ENJAYMO if you are allergic to sutimlimab-jome or any of the ingredients in ENJAYMO.
ENJAYMO can cause serious side effects, including:
The most common side effects of ENJAYMO include:
These are not all the possible side effects of ENJAYMO. Call your doctor for medical advice about side effects.
Before receiving ENJAYMO, tell your healthcare provider about all of your medical conditions, including if you:
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
Please see full Prescribing Information, including Medication Guide.
About cold agglutinin disease
CAD is a chronic and rare blood disorder that impacts the lives of an estimated 5,000 people in the US It is caused by antibodies called cold agglutinins binding to the surface of red blood cells, which starts a process that causes the body’s immune system to mistakenly attack healthy red blood cells and cause their rupture (hemolysis). Red blood cells have the vital job of carrying oxygen throughout the body, so people with CAD may experience severe anemia, which can result in fatigue, weakness, shortness of breath, light-headedness, chest pain, irregular heartbeat, and other potential complications.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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