Treatment with Cerezyme
People with Type 1 Gaucher disease are deficient in the enzyme glucocerebrosidase. This enzyme is responsible for breaking down a certain fat molecule called glucocerebroside. This causes a buildup of glucocerebroside in certain cells, called Gaucher cells.
Cerezyme replaces the missing enzyme in Type 1 Gaucher disease. Cerezyme is a modified form of glucocerebrosidase, created using recombinant DNA technology.
Cerezyme therapy has been used since 1994 in thousands of patients around the world. In particular, Cerezyme has been used to treat Type 1 Gaucher patients who have the following conditions:
- Anemia (low red blood cell count)
- Low platelet count
- Spleen enlargement
- Liver enlargement
- Bone disease
Cerezyme therapy has been shown to help reduce, relieve or reverse many of the signs and symptoms of Type 1 Gaucher disease. However, experiences may vary from one person to the next. To find out more about the possible effects of Cerezyme therapy, see Cerezyme Effects.
How does Cerezyme work?
Cerezyme acts like a naturally occurring enzyme, glucocerebrosidase, and targets cells that have a build up of glucocerebroside (Gaucher cells). Cerezyme works inside the lysosome, a specialized compartment of the cell, where the breakdown of glucocerebroside (a fat molecule) normally occurs. Glucocerebroside is broken down into glucose and ceramide. This can then be removed from the cell by natural processes. If you stop taking Cerezyme, glucocerebroside will reaccumulate and symptoms can return. Cerezyme therapy is prescribed because it has been shown to help reduce, relieve or reverse certain signs and symptoms of Type 1 Gaucher disease.
What is the dose and how is it administered?
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.
What are the possible side effects associated with Cerezyme therapy?
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.
To learn more, please see full product information; contact Genzyme at 1-800-745-4447.
What changes to my condition may occur?
Cerezyme has been shown to help reduce, relieve or reverse certain signs and symptoms of Type 1 Gaucher disease. During clinical studies, improvements were seen in patients’ hemoglobin levels and platelet counts (specific blood tests) and reductions in liver and spleen sizes within the first 6 months. Treatment experiences may vary from one person to the next. While some signs and symptoms may improve within months, others may take years; in patients with more advanced disease, some may not be improved at all.
Should I stop taking my other medications while on Cerezyme?
Therapy with Cerezyme does not mean you should stop using your other medications. You should not change your medication unless directed to do so by your doctor.
How long will I need to take Cerezyme?
Cerezyme is not a cure for Type 1 Gaucher disease; that is, it does not correct the underlying genetic defect. Because your body does not produce sufficient amounts of enzyme on its own or the enzyme is not active, Cerezyme is expected to be a long-term part of your Type 1 Gaucher disease therapy. To continue to benefit from treatment, you should receive regular intravenous infusions, even though you may feel “better”. This is because, once therapy stops, Gaucher cells may build up again and symptoms can return.
What should I tell my doctor before I start Cerezyme?
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. Pregnancy or breast-feeding: It is not known whether Cerezyme can cause fetal harm when administered to a pregnant woman or can affect the ability to get pregnant. Cerezyme should not be administered during pregnancy except when the indication and need are clear and the potential benefit is judged by your doctor to substantially justify the risk. If you think you are pregnant or if you plan to become pregnant, your doctor needs to know as soon as possible. Also, tell your doctor if you are breastfeeding. It is not known whether Cerezyme is present in the mother’s milk. Because many drugs are present in the mother’s milk, caution should be exercised when Cerezyme is administered to a nursing woman. Medical problems or allergies: Be sure your doctor is aware of any medical problems or allergies you have now or have had in the past even if they are not related to Type 1 Gaucher disease. Other medications: Talk to your doctor about any medications you are taking, including over-the-counter medicines and dietary supplements (vitamins).
Who should not receive Cerezyme?
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.
Is information on Gaucher disease and the use of Cerezyme being collected?
A Gaucher Registry has been established. 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. In the 17 years that the Registry has been collecting data it has added significantly to the understanding of Gaucher disease, providing treating doctors with information on treatment guidelines, monitoring recommendations, and consensus statements. Speak to your doctor about participating in this registry.
Where can I learn more about Genzyme Support Programs?
Genzyme offers a complete network of programs and services for patients, their families, and healthcare providers. These support services were developed to provide resources and information to help patients advocate for their comprehensive care. For information about educational resources, health insurance, physicians with experience in the management of Gaucher disease, diagnostic testing, or other programs, please contact Genzyme at 617-768-9000 or toll-free 800-745-4447 (option 2)
Indication & Usage
Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease 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)
Important Safety Information
Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients. Symptoms suggestive of allergic reaction happened in approximately 7% of patients, and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.
High blood pressure in the arteries of the lungs (pulmonary hypertension) and pneumonia have been observed in less than 1% of patients during treatment with Cerezyme. These are also known complications of Gaucher disease regardless of treatment. If you experience symptoms such as shortness of breath or chest pain, with or without fever, contact your doctor.
Approximately 14% of patients have experienced side effects related to treatment with Cerezyme. Some of these reactions occur at the site of injection such as discomfort, itching, burning, swelling or uninfected abscess. Other side effects, each of which was reported by less than 2% of patients, include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Temporary swelling in the legs has also been observed with drugs like Cerezyme.
Please see Full Prescribing Information (PDF).