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... View more

Bone Studies

Cerezyme is the ONLY ERT (enzyme replacement therapy) that has shown long-term efficacy and safety in multiple studies over 10 years and has been prescribed for over 20 years.1-3

View Indications and Usage

Choose Cerezyme: Improved bone mineral density (BMD), bone pain, and bone crises5-7

An overview: Cerezyme studies for Gaucher-related bone disease


*The Cerezyme treatment group from the Gaucher Registry analyses represents patients who received either alglucerase or imiglucerase.
Percentage changes from baseline were calculated by dividing the change from baseline by the baseline value and multiplying by 100.

Prospective, 48-Month Study5

Study design (Sims et al)5

View the full study design for complete details, including inclusion criteria and outcome measures

Long-term use of Cerezyme improved BMD5

By month 48, lumbar spine BMD in Cerezyme patients had reached near-normal levels

Long-term use of Cerezyme improved BMD

Cerezyme decreased
the occurrence of
bone pain and bone
crises5

Cerezyme reduced bone pain and bone crises over 48 months

-34% reduction in the number of patients with bone pain within 6 months
-85% reduction in the number of patients with bone crisis within 12 months

* Percentage changes from baseline were calculated by dividing the change from baseline by the baseline value and multiplying by 100.

Safety from Sims et al5

  • The most common AEs were chills, flushing, and arthralgia, each reported in 4 patients (12%)
  • One patient withdrew from the study because of a severe infusion reaction, including anxiety, chest pain, hypertonia, chills, tachycardia, and vomiting, from which he recovered without sequelae

Retrospective ICGG Registry studies

8-year ICGG Registry analysis (Wenstrup et al): Study parameters6

An observational, retrospective analysis in adults (men aged 18 to 70 years, women aged 18 to 50 years) enrolled in the ICGG Gaucher Registry for whom lumbar spine BMD measurements were available. Read more...

Cerezyme patients achieved near-normal BMD after 8 years6

DXA Z-score in patients with no Cerezyme
Cerezyme patients achieved near-normal BMD after 8 years

* Percentage changes from baseline were calculated by dividing the change from baseline by the baseline value and multiplying by 100.

  • Cerezyme dosing should be individualized to each patient
  • Initial dosages range from 2.5 U/kg of body weight 3 times a week to 60 U/kg once every 2 weeks; 60 U/kg every 2 weeks is the dosage for which the most data are available
  • Dosage adjustments should be made on an individual basis and may increase or decrease based on achievement of therapeutic goals as assessed by routine comprehensive evaluations of the patient’s clinical manifestations

4-year ICGG Registry analysis (Charrow et al): Study parameters7

This retrospective analysis used data from the ICGG Gaucher Registry on patients with bone crisis and/or bone pain data for 1 year prior to Cerezyme, and each year for 3 years after the start of Cerezyme. The year before treatment was considered to be baseline. Read more...

Cerezyme significantly decreased the occurrence of bone pain and bone crises7

-39% reduction in the number of patients with bone pain within 12 months
-71% reduction in the number of patients with bone pain within 12 months

*Percentage changes from baseline were calculated by dividing the change from baseline by the baseline value and multiplying by 100.

    References:
  1. Cerezyme [prescribing information]. Cambridge, MA: Genzyme Corporation; 2018.
  2. Weinreb N, Taylor J, Cox T, Yee J, vom Dahl S. A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase. Am J Hematol. 2008;83(12):890-895.
  3. Weinreb NJ, Goldblatt J, Villalobos J, et al. Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment. J Inherit Metab Dis. 2013;36(3):543-553.
  4. Grabowski GA, Barton NW, Pastores G, et al. Enzyme therapy in type 1. Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med. 1995;122(1):33-39.
  5. Sims KB, Pastores GM, Weinreb NJ, et al. Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48-month longitudinal cohort study. Clin Genet. 2008;73(5):430-440.
  6. Wenstrup RJ, Kacena KA, Kaplan P, et al. Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease. J Bone Miner Res. 2007;22(1):119-126.
  7. Charrow J, Dulisse B, Grabowski GA, Weinreb NJ. The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease. Clin Genet. 2007;71(3):205-211.
  8. Andersson H, Kaplan P, Kacena K, Yee J. Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics. 2008;122(6):1182-1190.
  9. Weinreb NJ, Kaplan P. The history and accomplishments of the ICGG Gaucher registry. Am J Hematol. 2015;90(suppl 1):s2-s5.
  10. Starzyk K, Richards S, Yee J, Smith SE, Kingma W. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Mol Genet Metab. 2007;90(2):157-163.
An established ERT for children
An established ERT for children

Efficacy in key disease parameters in pediatric patients with Gaucher disease type 1

Explore pediatric data
When an ERT is needed
When an ERT is needed

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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 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 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 Sanofi Genzyme Medical Information at 1-800-745-4447, Option 2.

Please see Full Prescribing Information (PDF).