Recordati Rare Diseases Announces Real-World Data on Long-Term Safety of ENJAYMO® (sutimlimab-jome) in Cold Agglutinin Disease and Cold Agglutinin Syndrome (CAD/CAS) at American Society of Hematol...

Results from CADENCE, the first and largest multinational prospective registry for patients with CAD/CAS, describe safety events observed in patients treated with sutimlimab, with an average treatment duration of more than two years

ENJAYMO® is approved for treatment of hemolysis (breakdown of red blood cells) in CAD, and is not approved for use in patients with CAS

BRIDGEWATER, N.J., Dec. 9, 2025 /PRNewswire/ -- Recordati Rare Diseases Inc. today announced the presentation of real-world data that build upon the safety data of sutimlimab, an FDA-approved therapy for hemolysis in adults with cold agglutinin disease (CAD). In a poster presentation at the 67^th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla., researchers presented an updated analysis from the CADENCE registry¹, the first multinational, prospective, longitudinal registry for patients with CAD and cold agglutinin syndrome (CAS), two rare types of autoimmune hemolytic anemia (AIHA). As acknowledged by the FDA-approved labeling of sutimlimab-jome, the safety and efficacy of sutimlimab has not been established in CAS.

"The CADENCE registry comprises the world's largest dataset of treatment patterns and regimens, including a sutimlimab-treated cohort, in patients with cold agglutinin disease and cold agglutinin syndrome, offering a unique, real-world view of how patients respond to long-term therapy," said lead investigator Catherine M. Broome, MD, Professor of Medicine and Director of Cellular Apheresis at the Lombardi Cancer Center at MedStar Georgetown University in Washington, DC. "Our updated analysis of the sutimlimab cohort did not identify any new safety signals after an average treatment duration of more than two years. The safety findings were consistent with earlier clinical trial data in CAD."

CADENCE is an observational registry; results are descriptive and should be interpreted with caution.

Dr. Broome and colleagues assessed long-term safety data from the 63 sutimlimab-treated patients from the US, Japan, and Europe who were enrolled in the 442-patient CADENCE registry between December 12, 2019, and February 4, 2025. The sutimlimab cohort included 58 patients who were diagnosed with CAD, four with CAS, and one with an unconfirmed diagnosis. The mean (standard deviation [SD]) age of sutimlimab-treated patients was 72.7 (8.8) years at the time of enrollment and the proportion of female patients was 76.2%. At the time of data cut-off, the median (Q1, Q3) and mean (SD) sutimlimab treatment duration was 22.7 (10.4, 32.6) and 25.1 (18.7) months, respectively. Sutimlimab is not approved in CAS.

A total of 159 adverse events (AEs) were reported in 33 (52.4%) of the sutimlimab-treated patients. Six (9.5%) patients experienced AEs that the investigator considered related to sutimlimab. Of the 37 serious adverse events (SAEs) reported in 11 (17.5%) patients, one SAE -- chest pain/stable angina -- was considered an unexpected sutimlimab-related SAE. There was one reported death in the cohort, due to low right lung pneumonia; this SAE was considered unrelated to sutimlimab.

AEs of special interest (AESIs) in sutimlimab-treated patients included serious infections in three patients, and arterial hypertension (reported as non-serious) in two patients; three patients who experienced AESIs had concomitantly used rituximab; none of these AESIs were considered related to sutimlimab. Non-serious AESIs of acrocyanosis were reported in eight patients, two of which were considered possibly related to sutimlimab. Please see Important Safety Information farther down in this press release.

CADENCE is an observational registry; the results reported at ASH are descriptive and should be interpreted with caution. ENJAYMO® is not approved for the treatment of CAS.

"The CADENCE registry data expand upon the real-world evidence base supporting the use of sutimlimab therapy for patients with cold agglutinin disease," commented Alessandro Albuquerque, MD, PhD, Chief Medical Officer and Vice President of Medical Affairs and Clinical Development at Recordati. "Recordati remains committed to meeting the needs of individuals living with rare disorders, and we look forward to additional updates from the CADENCE registry as follow-up continues."

The underlying research referenced in this press release, including the referenced presentation, and their contributing authors were financially supported by Recordati Rare Diseases.

About Cold Agglutinin Disease (CAD)

Cold agglutinin disease (CAD) is a rare type of autoimmune hemolytic anemia (AIHA), characterized as a low-grade lymphoproliferative disorder of the bone marrow.² In CAD, autoantibodies bind to erythrocytes at temperatures ≤37°C, leading to complement-mediated hemolysis. CAD symptoms include severe, debilitating fatigue and other clinical manifestations (e.g., dyspnea, tachycardia) that can impact patients' quality of life. While the median age of onset is approximately 60 years, CAD has been diagnosed in patients as young as 30.

About ENJAYMO® (sutimlimab-jome)  

ENJAYMO® is a humanized monoclonal antibody that is designed to specifically bind to complement protein component 1, s (C1s) subcomponents in the C1 complement complex, which is part of the classical complement pathway. By binding to C1s, Enjaymo inhibits activation of classical complement pathway, preventing deposition of complement opsonins on red blood cells and resulting in inhibition of hemolysis. ENJAYMO® does not inhibit the lectin and alternative pathways of the complement system. ENJAYMO® was approved by the US Food and Drug Administration (FDA) in February 2022 for the treatment of hemolysis in adults with CAD. The Japanese Ministry of Health, Labor and Welfare approved ENJAYMO® in June 2022. The European Medicines Agency (EMA) granted marketing authorization to ENJAYMO® in November 2022. Both the FDA and the EMA have designated ENJAYMO® an orphan drug.

What is ENJAYMO?

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:

• Serious Infections: ENJAYMO is a prescription medicine that affects your immune system. ENJAYMO may lower the ability of your immune system to fight infections. ENJAYMO increases your chance of getting serious infections including those caused by encapsulated bacteria, including Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or cause death if not recognized and treated early.
  • You must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of ENJAYMO.
  • If your healthcare provider decides that urgent treatment with ENJAYMO is needed, you should receive vaccinations as soon as possible.
  • If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting ENJAYMO. Your healthcare provider will decide if you need additional vaccines.
  • Vaccines do not prevent all infections caused by encapsulated bacteria. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious infection: fever with or without shivers or chills, fever with chest pain and cough, fever with high heart rate, headache and fever, confusion, clammy skin, fever and a rash, fever with breathlessness or fast breathing, headache with nausea or vomiting, headache with stiff neck or stiff back, body aches with flu-like symptoms, and/or eyes sensitive to light.
• 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, decrease in blood pressure, chest discomfort, rapid heartbeat, nausea, injection site reaction, flushing, headache, dizziness, rash, and itchy skin.
• 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, and 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: tiredness, shortness of breath, rapid heart rate, and blood in your urine or dark urine.

The most common side effects of ENJAYMO include: increase in blood pressure, urinary tract infection, respiratory tract infection, bacterial infection, swelling in lower legs or hands, joint pain, headache, nausea, runny nose, bluish color to the lips and skin, dizziness, feeling tired or weak, cough, and changes in color or sensation in the fingers and toes (Raynaud's phenomenon).

These are not all the possible side effects of ENJAYMO. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Call 1-800-FDA-1088 or visit www.fda.gov/medwatch.

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, 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.

Please see Full Prescribing Information, including Medication Guide.

About Recordati Rare Diseases

Recordati Rare Diseases is Recordati's dedicated business unit focused on Rare Diseases. Recordati is an international pharmaceutical Group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas, and APAC, with over 4,500 employees.

Recordati Rare Diseases' mission is to reduce the impact of extremely rare and devastating diseases by providing urgently needed therapies. We work side-by-side with rare disease communities to increase awareness, improve diagnosis, and expand availability of treatments for people with rare diseases.

Recordati Rare Diseases Inc. is the company's U.S. corporate headquarters located in Bridgewater, NJ, with the global headquarter offices located in Milan, Italy. 

For full prescribing information, including approved indications and important safety information about marketed products, please visit: https://recordatirarediseases.us/our-products/.

¹ Röth A, Michel M, Barcellini W, et al. Real-world safety of sutimlimab in patients with CAD/CAS: a multinational, multicenter, observational, prospective cohort study. Presented at 67^th American Society of Hematology (ASH) Annual Meeting and Exposition, Orlando, Fla., December 8, 2025. Poster #6242.
² Berentsen S, D'Sa S, Randen U, Malecka A, Vos JMI. Cold agglutinin disease: improved understanding of pathogenesis helps define targets for therapy. Hemato. 2022;3(4):574-594. doi: 10.3390/hemato3040040.

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