Gaucher disease is caused by an inherited genetic defect. People with Gaucher disease are deficient in the enzyme glucocerebrosidase, which is responsible for breaking down a certain fat molecule called glucocerebroside. This causes a buildup of glucocerebroside in certain cells, called Gaucher cells. To learn more about Gaucher disease, visit the About Gaucher Disease area.

It is now possible to replace the missing enzyme in Type 1 Gaucher disease. Cerezyme^® (imiglucerase for injection) 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.

Cerezyme therapy

Cerezyme therapy has helped thousands of Gaucher patients. In particular, Cerezyme has been used to treat Type 1 Gaucher patients who have the following conditions:

Anemia

Low platelet count

Spleen enlargement

Liver enlargement

Bone disease

For more information, please see the important safety information below.

How does Cerezyme work?

Cerezyme acts like naturally occurring glucocerebrosidase and targets Gaucher cells. Inside the cells, Cerezyme breaks up glucocerebroside into smaller components glucose and ceramide. These smaller components can then be removed from the cell by natural processes, just as they are in people without Gaucher disease.

Cerezyme delivery

Cerezyme therapy is given intravenously over a couple of hours. This may be done in a variety of settings, such as a doctor’s office, a treatment center, or home. To learn more about treatment with Cerezyme, take a look at the What to Expect from Therapy. In addition, please see the Full Product Information (PDF)

Dosage

The initial dose of Cerezyme therapy is based on each patient’s weight and the severity of his or her symptoms. The doctor will perform a comprehensive evaluation and will determine the most appropriate dose for you or your child. Using the tests described in Monitoring Gaucher Disease, the doctor can monitor the disease and individualize the dosage depending on the response to ERT.

Important Safety Information

Side effects related to Cerezyme® (imiglucerase for injection) 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, vomiting, abdominal pain, 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. Symptoms suggestive of allergic reaction 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. Approximately 15% of patients have developed immune responses (antibodies); periodic monitoring by your physician is suggested. Patients should notify their physician immediately if they experience any side effects with treatment. Cerezyme is available by prescription only. For more information, consult your physician. To learn more, please see the full product information (PDF) or contact Genzyme at 1-800-745-4447.