This site is currently under construction. The information below is meant to serve as a general overview of Gaucher disease and Cerezyme. For additional information, speak to a doctor or contact the Sanofi Genzyme CareConnectPSS team by completing a request information form, calling 1-800-745-4447 (Option 3) Monday through Friday, 8:00 AM to 6:00 PM EST, or visiting CareConnectPSS.com.
Gaucher (pronounced go-shay) disease is a rare, progressive, inherited condition that causes a fatty substance, called glucosylceramide (gloo-ko-sil-sara-mide, also called GL-1) to build up in certain organs or bones. As GL-1 builds up, people with Gaucher disease may experience excessive bruising and bleeding, and protruding abdomens caused by swelling of the liver and/or spleen.
It is the buildup of GL-1 that causes the signs and symptoms of Gaucher disease, but some patients may not have any symptoms at all despite disease progression.
There are 3 types of Gaucher disease (Types 1, 2, and 3), more than 90% of patients have type 1 making it the most common. Gaucher disease type 1 can be effectively managed once a diagnosis is made. The goal of treatment is to reduce or prevent the buildup of GL-1. Treatment for Gaucher disease type 1 not only helps alleviate certain symptoms, but can also help reduce the chance of more severe or permanent changes. The 2 treatment approaches available are substrate reduction therapy (an oral medicine) and enzyme replacement therapy (ERT).
Treatment for Gaucher disease type 1 not only helps alleviate certain symptoms, but can also help reduce the chance of more severe or permanent changes. The goal of treatment is to reduce the accumulation of GL-1. The 2 treatment approaches available are substrate reduction therapy (an oral medicine) and enzyme replacement therapy (ERT).
The ONLY ERT that demonstrated long-term efficacy and safety in multiple studies and has over 20 years of real-world experience.
Cerezyme is an enzyme replacement therapy (ERT), indicated for the long-term treatment of pediatric and adult patients with Gaucher disease type 1 that results in one or more of the following conditions: anemia (low red blood cell count), thrombocytopenia (low blood platelet count), bone disease, hepatomegaly or splenomegaly (enlarged liver or spleen). Cerezyme helps your body break down the excess GL-1. If you stop or miss treatment GL-1 may re-accumulate and symptoms can return.
The ERT chosen by physicians for patients with Gaucher disease type 1 worldwide since 1994
Proven efficacy with demonstrated improvements in spleen, liver, hemoglobin, platelet counts and certain bone parameters
Long-term efficacy supported by adult and pediatric data in International Collaborative Gaucher Group (ICGG) Gaucher Registry analyses
Approximately 13.8% of patients reported adverse events (AEs)
Supported by 10-year data from an international safety monitoring database
Your doctor will determine your Cerezyme dose and frequency. Dose should be individualized to each patient and is based on weight and disease severity. Cerezyme is given by intravenous (IV) infusion, meaning that the medication is delivered directly into your bloodstream. An infusion of Cerezyme typically takes 1-2 hours. It is generally administered every two weeks. If you miss an infusion, talk to your doctor about whether you need to make up the dose.
Approximately 15% of patients have developed immune responses (antibodies). These patients have a higher risk of an allergic reaction (hypersensitivity). Use Cerezyme® (imiglucerase for injection) carefully if you have had an allergic reaction to the product in the past. Symptoms suggestive of allergic reaction happened in 6.6% of patients, and include anaphylactoid reaction (a serious allergic reaction), itching, flushing, hives, an accumulation of fluid under the skin, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure.
Side effects related to Cerezyme administration have been reported in less than 15% of patients. Each of the following events occurred in less than 2% of the total patient population. Reported side effects include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Because Cerezyme therapy is administered by intravenous infusion, reactions at the site of injection may occur: discomfort, itching, burning, swelling or uninfected abscess. Cerezyme is available by prescription only. Patients should notify their physician immediately if they experience any side effects while undergoing treatment with Cerezyme. For more information, consult your physician.
Therapy with Cerezyme does not mean you should stop using other medication. You should tell your doctor or pharmacist if you are using, have used recently, or might use any other medicine, including over-the-counter medicines and herbal supplements, while taking Cerezyme. You should not change your medication unless directed to do so by your doctor. Your doctor will work with you to determine the appropriate course of treatment.
Talk to your doctor about any of the following situations that apply to you, as they may affect how your doctor will choose to treat you.
There are no known contraindications (a specific situation where a drug, procedure, or surgery should not be used, because it may be harmful to the patient) to the use of Cerezyme. Your doctor should carefully re-evaluate treatment if you experience a significant allergic reaction while taking Cerezyme. The safety and effectiveness of Cerezyme in patients younger than 2 years have not been established.
Sanofi Genzyme's dedicated team of professionals is available to provide disease education and help address your needs, including assistance with health insurance issues, all free of charge.
CareConnectPSS represents Sanofi Genzyme’s more than 25-year commitment to supporting the rare disease community. CareConnectPSS is designed to support each patient’s unique journey through dedicated Case Managers and Patient Education Liaisons (also called “PELs”) who provide one-on-one assistance through a range of resources, programs, and other support offerings. Whether you are newly diagnosed or already receiving treatment, your CareConnectPSS team will work closely with you and your health care providers to give you the confidential and personalized support you need.
Programs and other offerings on a range of disease, treatment,and support topics
Dedicated CareConnectPSS Case Managers and Patient Education Liaisons
Disease-specific information, including genetic education andother resources
Care coordination for treatment-site changes, home infusions, moves, vacations, and more
Assistance with understanding new or changing insurance, as well as resources tohelp with out-of-pocket costs
CareConnectPSS also includes a Cerezyme Support Communication Program to help patients adhere to their treatment plan. Communications to Cerezyme patients include:
Fill out our request information form to get more details on disease education, treatment information, financial support, and patient support.
Access to these and other services is voluntary, and patients are not obligated to begin treatment if they contact us. You and your physician will make all treatment-related decisions, and, most importantly, the privacy and security of your personal information are always protected.
There are various professional medical organizations, national and international Gaucher disease groups, Gaucher disease education websites, and patient assistance programs that work to support Gaucher disease patients and their families and caregivers. To view a list of these organizations click here.
All information and resources provided by these organizations are operated and maintained by the specified entry. Please note this list is meant to serve as a general reference and additional resources may also be available through other organizations. With the exception of specific Sanofi Genzyme* resources, Sanofi Genzyme does not endorse any particular organization or the content and programs they offer.
The ICGG Registry is the world’s largest cooperative, observational database on Gaucher disease. The Gaucher Registry is sponsored by Sanofi Genzyme and directed by the ICGG, a group of physicians who are experts in the management of Gaucher disease. The Gaucher Registry is an observational database intended to explore and define the natural history of Gaucher disease as well as track and characterize patient response to therapy. The data the registry has been collecting has added significantly to the understanding of Gaucher disease, providing treating doctors with information on treatment guidelines, monitoring recommendations, and consensus statements. Physicians can learn more about the Gaucher Registry by clicking here.
Understanding your insurance coverage is very important. Our CareConnectPSS Case Managers are here to help you better understand your insurance benefits so you can get access to the treatment you need. Sanofi Genzyme offers 2 types of access programs for eligible patients.
The CareConnectPSS Co-Pay Program helps eligible, US patients who are prescribed one of Sanofi Genzyme’s treatments pay for eligible, out-of-pocket, drug-related expenses, including copays, coinsurance, and deductibles.
The program is open to individuals who:
The program is not valid for prescriptions eligible to be reimbursed, in whole or in part, by Medicaid, Medicare (including Medicare Part D), or other federal or state programs (including any state prescription drug assistance programs).
Complete an online enrollment application form. Your application will be reviewed for eligibility. If you are eligible, you will be automatically enrolled in the program. If eligible, you will receive your copay card within 7-10 days
For questions or to learn more about the program, call your CareConnectPSS Case Manager at this 1-800-745-4447, option 3
*No claim for reimbursement of any out-of-pocket expense covered by the Copay Program may be submitted to any third-party payer, where public or private. The Copay Program is available only in the United States and cannot be combined with any other rebate/coupon, free trial, or similar offer. Copay benefits are not transferable. The Copay Program assists patients with their out-of-pocket drug costs of their Sanofi Genzyme therapy treatment only and does not cover or provide support for the cost of MD office visits/evaluations, nursing services/observation periods, blood work, x-rays or other testing, pre-medications/other medications, EpiPens, transportation or other related services. Sanofi Genzyme reserves the right to make eligibility decisions, set program maximums, and rescind, revoke, or amend this program without notice.
The Sanofi Genzyme Charitable Access Program (CAP) has been established in the United States through the Genzyme Charitable Foundation, Inc. The program is committed to providing Cerezyme to individuals who:
Qualified individuals with Gaucher disease type 1 whose physicians have recommended treatment with Cerezyme may be eligible for the Charitable Access Program. If you are ineligible for our program, your CareConnectPSS Case Manager will work with you and your health care providers to explore alternative coverage options.
To be considered for the program, you will be asked to provide the following:
Your CareConnectPSS Case Manager will coordinate with you and your physician to help obtain the necessary documentation and will keep you updated on the status of your application.
Applications are reviewed on a monthly basis and are kept confidential by the Sanofi Genzyme Charitable Access Program Committee.
Please note that the Sanofi Genzyme Charitable Access Program is considered a temporary program. Patients and their families are expected to continue exploring alternative resources with the assistance of a CareConnectPSS Case Manager. These may include:
The Sanofi Genzyme Charitable Access Program may be discontinued at any time at the discretion of the Sanofi Genzyme Charitable Access Program Committee.