FDA Approves Enjaymo (sutimlimab-jome) for Use in Patients with Cold Agglutinin Disease
Paris, February 4, 2022. -- The U.S. Food and Drug Administration (FDA) has approved Enjaymo™ (sutimlimab-jome) to decrease the need for red blood cell transfusion due to hemolysis in adults with cold agglutinin disease (CAD). Enjaymo is the first and only approved treatment for people with CAD and works by inhibiting the destruction of red blood cells (hemolysis).
Executive Vice President, Head of Specialty Care
“Until now, people living with cold agglutinin disease haven’t had an approved treatment option to manage the constant destruction of red blood cells. Without healthy, viable red blood cells, a chain reaction of debilitating signs and symptoms can be triggered, starting with severe anemia. Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start.”
CAD, a rare autoimmune hemolytic anemia, 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). As red blood cells have the vital job of carrying oxygen throughout the body, patients 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. CAD is a chronic and rare blood disorder that impacts the lives of an estimated 5,000 people in the U.S.
Enjaymo, targeting C1s in the classical complement pathway
Enjaymo is a humanized monoclonal antibody that is designed to selectively target and inhibit C1s in the classical complement pathway, which is part of the innate immune system. By blocking C1s, Enjaymo inhibits the activation of the complement cascade in the immune system and inhibits C1-activated hemolysis in CAD to prevent the abnormal destruction of healthy red blood cells. Enjaymo does not inhibit the lectin and alternative pathways.
Enjaymo Phase 3 pivotal CARDINAL study results supporting approval
The approval of Enjaymo in the U.S. is based on positive results from the 26-week open label, single arm pivotal Phase 3 study in patients with CAD (n=24) who have a recent history of blood transfusion, also known as the CARDINAL study.
Catherine Broome, MD
Associate professor of medicine at Georgetown University Lombardi Comprehensive Cancer Center, and a principal investigator in the CARDINAL study
"For people living with cold agglutinin disease, it is as if their body’s immune system is waging a war on itself. The relentless destruction of healthy red blood cells is a daily, silent reality for people with CAD. For the first time, we have a treatment that targets complement-mediated hemolysis, which is the underlying cause of the red blood cell destruction in many CAD patients. In the pivotal study, patients treated with sutimlimab had an improvement in anemia as measured by hemoglobin and bilirubin levels during the 26-week study.”
In the study, Enjaymo met its primary efficacy endpoint, which was a composite endpoint defined as the proportion of patients who achieved normalization of hemoglobin (Hgb) level ≥12 g/dL or demonstrated an increase from baseline in Hgb level ≥2 g/dL at the treatment assessment time point (mean value from weeks 23, 25, and 26) and no blood transfusion from weeks 5 through 26 or medications prohibited per the protocol from weeks 5 through 26. Secondary endpoints were also met, including improvements in hemoglobin and normalization of bilirubin.
- The majority of patients (54%; n=13) met the composite primary endpoint criteria with 63% (n=15) of patients achieving a hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL; 71% (n=17) of patients remaining transfusion-free after week five; and 92% (n=22) of patients did not use other CAD-related treatments.
- For the secondary measures on disease process, patients enrolled experienced a mean increase in hemoglobin level of 2.29 g/dL (SE: 0.308) at week 3 and 3.18 g/dL (SE: 0.476) at the 26-week treatment assessment timepoint from the mean baseline level of 8.6 g/dL. The mean reduction in bilirubin levels (n=14) was by -2.23 mg/dL (95% CI: -2.49 to -1.98) from a mean baseline level of 3.23 mg/dL (2.7-fold ULN).
In the CARDINAL study, the most common adverse reactions occurring in 10 percent 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 percent (3/24) of patients who received Enjaymo. These serious adverse reactions were streptococcal sepsis and staphylococcal wound infection (n=1), arthralgia (n=1), and respiratory tract infection (n=1). None of the adverse reactions led to discontinuation of Enjaymo in the study. Dosage interruptions due to an adverse reaction occurred in 17 percent (4/24) of patients who received Enjaymo.
Following the completion of the 26-week treatment period of CARDINAL (Part A), eligible patients continued to receive Enjaymo in an extension study.
The recommended dose of Enjaymo is based on body weight (6,500 mg for people 39-75 kg and 7,500 mg for people >75 kg). Enjaymo is administered intravenously weekly for the first two weeks with administration every two weeks thereafter.
Enjaymo is expected to be available in the U.S. in the coming weeks. The U.S. list price, or wholesale acquisition cost, of Enjaymo is $1,800 per vial. Actual costs to patients are generally anticipated to be lower as the list price does not reflect insurance coverage, copay support, or financial assistance from patient support programs. As part of our commitment to ensure treatment access and affordability for innovative therapies, Enjaymo Patient Solutions provides disease education, financial and co-pay assistance programs and other support services to eligible patients. For more information, please call 1-833-223-2428.
Enjaymo received FDA Breakthrough Therapy and Orphan Drug designation, and priority review, which is reserved for medicines that, if approved, would represent significant improvements in safety or efficacy in treating serious conditions. Outside of the U.S., sutimlimab has been submitted to regulatory authorities in Europe and Japan and reviews are ongoing.
Enjaymo is a prescription medicine used to decrease the need for red blood cell transfusion due to 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:
- Serious Infections: Enjaymo is a prescription medicine that affects your immune system. Enjaymo can lower the ability of your immune system to fight infections. People who take Enjaymo may have an increased risk of getting infections caused by certain kinds of bacteria such as Neisseria meningitides, Streptococcus pneumoniae, and Haemophilus influenzae. These infections may be serious or life-threatening. Some infections may quickly become life-threatening or cause death if not recognized and treated early.
- You need to receive vaccinations against infections caused by certain kinds of bacteria at least 2 weeks before your first dose of Enjaymo. You may need to have additional vaccinations during treatment
- If your healthcare provider decides that urgent treatment with Enjaymo is needed, you should receive vaccinations as soon as possible.
- Vaccinations may reduce the risk of these infections, but do not prevent all infections. Call your healthcare provider or get medical help right away if you get any new signs and symptoms of an infection, including:
- severe headache with stiff neck or back
- pain during urination or urinating more often than usual
- cough or difficulty breathing
- flu-like symptoms
- pain, redness or swelling of the skin
- Infusion-related reactions: Treatment with Enjaymo may cause infusion-related reactions, including allergic reactions that may be serious or life-threatening. Your healthcare provider may slow down or stop your Enjaymo infusion if you have an infusion-related reaction, and will treat your symptoms if needed. Tell your healthcare provider right away if you develop symptoms during your Enjaymo infusion that may mean you are having an infusion-related reaction, including:
- shortness of breath
- rapid heartbeat
- Risk of autoimmune disease: Enjaymo may increase your risk for developing an autoimmune disease such as systemic lupus erythematosus (SLE). Tell your healthcare provider and get medical help if you develop any symptoms of SLE, including:
- joint pain or swelling
- rash on the cheeks and nose
- unexplained fever
- If you have CAD and you stop receiving Enjaymo, your healthcare provider should monitor you closely for return of your symptoms after you stop Enjaymo. Stopping Enjaymo may cause the breakdown of your red blood cells due to CAD to return. Symptoms or problems that can happen due to red blood cell breakdown include:
- shortness of breath
- rapid heart rate
- blood in your urine or dark urine
The most common side effects of Enjaymo include:
- respiratory tract infection
- viral infection
- joint pain
- joint inflammation (arthritis)
- swelling of the lower legs, ankles, and feet
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:
- have a fever or infection, including a history of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C.
- have an autoimmune disease such as systemic lupus erythematosus (SLE), also known as lupus.
- are pregnant or plan to become pregnant. It is not known if Enjaymo will harm your unborn baby.
- are breastfeeding or plan to breastfeed. It is not known if Enjaymo passes into your breast milk.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
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Posted: February 2022