Research and Development

Genzyme is committed to developing therapies for Gaucher disease and other rare genetic disorders. Through years of dedicated research, the company developed the first specific therapies for Type 1 Gaucher disease and other rare lysosomal storage diseases.

As experience with Cerezyme has increased, more information has become available upon which to make recommendations about treatment protocols and monitoring treatment efficacy. Genzyme’s ongoing clinical studies and the Genzyme-supported Gaucher Registry help answer questions such as how dosing affects treatment outcomes, and how Cerezyme affects the signs and symptoms of Gaucher disease over long periods of continued treatment.

In addition to these clinical studies, Genzyme is committed to advancing new approaches to the treatment of Gaucher disease.

Genzyme is planning and conducting a number of clinical trials involving patients with Gaucher disease or other lysosomal storage disorders. For more information about these trials, send a query on the Ask Us page, call Genzyme Medical Information at 800-745-4447, option 2 or 617-768-9000 or visit clinicaltrials.gov.

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
  2. thrombocytopenia
  3. bone disease
  4. hepatomegaly or splenomegaly

Important Safety Information

Approximately 15% of patients have developed IgG antibodies to Cerezyme during the first year of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity, and these patients have a higher risk of hypersensitivity. It is suggested that patients be monitored periodically for IgG antibody formation during the first year of treatment.

Hypersensitivity has also been observed in patients without detectable IgG antibodies. Symptoms suggestive of hypersensitivity have been noted in approximately 6.6% of all patients, and anaphylactoid reactions in less than 1%. Treatment with Cerezyme should be approached with caution in patients who have exhibited hypersensitivity symptoms such as pruritus, flushing, urticarial, angioedema, chest discomfort, dyspnea, coughing, cyanosis, and hypotension. Pre-treatment with antihistamines and/or corticosteroids and a reduced rate of infusion may allow continued treatment in most patients.

In less than 1% of patients, pulmonary hypertension and pneumonia have been observed during treatment with Cerezyme. These are known complications of Gaucher disease regardless of treatment. Patients with respiratory symptoms in the absence of fever should be evaluated for the presence of pulmonary hypertension.

Approximately 13.8% of patients have experienced adverse events related to treatment with Cerezyme. Some of these are injection site reactions such as discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of venipuncture. Additional adverse reactions that have been reported include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Transient peripheral edema has also been reported for this therapeutic class of drug.

To report suspected adverse reactions, contact Genzyme at 800-745-4447, option 2 or FDA at 800-FDA-1088 or http://www.fda.gov/Safety/MedWatch

Please see Full Prescribing Information (PDF).

Cerezyme has been demonstrated for over 17 years to be an effective treatment for Type 1 Gaucher disease in adults and children.