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 and relieve certain signs and symptoms of Type 1 Gaucher Disease.

  • In clinical trials, Cerezyme improved anemia (low red blood cell count), thrombocytopenia (low platelet count), reduced spleen and liver size, and decreased cachexia (a wasting syndrome that results in loss of weight, fatigue, weakness and significant loss of body mass that cannot be reversed nutritionally).
  • Additionally, it was observed in the Gaucher Registry that Cerezyme therapy helped reduce and relieve many signs and symptoms of Type 1 Gaucher disease, that included low red blood cell count, low platelet count, bone pain, and enlarged spleen and liver.

To learn more about how treatment with Cerezyme can affect Gaucher disease, please see Cerezyme Effects.
 

How does Cerezyme work?

Cerezyme acts like a naturally occurring enzyme, glucocerebrosidase, which is deficient in patients with Gaucher Disease Type 1, resulting in a build up of a fat molecule in certain 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.

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.

What changes to my condition may occur?

Cerezyme has been shown to help reduce or relieve 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 and 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 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.

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. 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.

Where can I learn more about Sanofi Genzyme Support Services?

Managing a genetic disease is a lifetime commitment. From diagnosis to treatment, our 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.

  • For newly diagnosed patients: We provide in-person education and disease-specific resources
  • For patients just starting treatment: Our insurance verification assistance also provides treatment location options and logistical troubleshooting as well as treatment-specific resources and answers to treatment-related questions
  • For patients who are traveling or moving: We can help locate treatment centers, doctors, and other resources at their destination
  • For patients who may be changing jobs: We provide insurance transition assistance
  • For those in need of assistance with family matters: We provide information on medical family trees, genetics testing, and diagnostic labs for patients who may be planning on starting a family or want more information for their siblings or relatives

Access to these and other services is voluntary, and you are not obligated to begin treatment if you contact us. You and your doctor make all treatment-related decisions, and most importantly the privacy and security of your personal situation is always protected.

For more information, contact Sanofi Genzyme Support Services at 800-745-4447 (option 3)

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:

  1. anemia (low red blood cell count)
  2. thrombocytopenia (low blood platelet count)
  3. bone disease
  4. 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).

You may report side effects to the FDA at 1-800-FDA-1088.

Reference 1. Data on file. Genzyme Corporation; Cambridge, MA.