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:
- anemia
- thrombocytopenia
- bone disease
- hepatomegaly or splenomegaly
Important Safety Information
Approximately 15% of patients have developed IgG antibodies, and these patients have a higher risk of hypersensitivity reaction. Therefore periodic monitoring is suggested; caution should be exercised in patients with antibodies or prior symptoms of hypersensitivity. Symptoms suggestive of hypersensitivity occurred in 6.6% of patients, and include anaphylactoid reaction, pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and hypotension.
Reactions 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 adverse events include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Adverse events associated with the route of administration include discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of injection.
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).