Important Safety Information: Warnings and Precautions: Hypersensitivity and Infusion-Associated Reactions: Hypersensitivity reactions, some of which are serious and include anaphylaxis have been reported. Hypersensitivity and other infusion-associated reactions have been reported during or shortly after infusion and include pruritus, flushing, urticaria, angioedema, chest discomfort... View more

Bone Studies

Cerezyme is the ONLY ERT (enzyme replacement therapy) that has shown long-term efficacy and safety in multiple studies over 20 years and has been prescribed for over 25 years.1-3

View Indication and Usage

Cerezyme: effect on bone mineral density (BMD), bone pain, and bone crises4-6

An overview: Cerezyme studies for bone manifestations in Gaucher disease type 1


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

Prospective, 48-Month Study4

Study design (Sims et al)4

Overview of Cerezyme studies for Gaucher-related bone disease
View the full study design for complete details, including inclusion criteria and outcome measures

Long-term effect of Cerezyme on BMD4

Long-term use of Cerezyme improved BMD

Long-term effect of Cerezyme on the occurrence of
bone pain and bone
crises4

-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 al4

  • 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

AE=adverse event; BMD=bone mineral density; DXA=dual-energy x-ray absorbtiometry.

Retrospective ICGG Gaucher Registry studies

8-year ICGG Gaucher Registry analysis (Wenstrup et al): Study parameters5

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 impact on BMD over 8 years5

DXA Z-score in patients with no Cerezyme
Cerezyme impact on BMD over 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 patient1
  • The recommended dosage of Cerezyme based upon disease severity ranges from 2.5 U/kg 3 times a week to 60 U/kg once every 2 weeks1
  • Titrate the dosage based on clinical manifestations of disease and therapeutic goals for the patient1

4-year ICGG Gaucher Registry analysis (Charrow et al): Study parameters6

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. Data on bisphophonate use were not available. Read more...

Effect of Cerezyme for injection on the occurrence of bone pain and bone crises over 4 years6

-38% reduction in the number of patients with bone pain within 12 months
-74% 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; 2021.
  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, Camelo JS, Charrow J, et al. Gaucher disease Type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Mol Genet Metab. 2021;132(2):100-111.
  4. 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.
  5. 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.
  6. 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.
  7. 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.
  8. Weinreb NJ, Kaplan P. The history and accomplishments of the ICGG Gaucher Registry. Am J Hematol. 2015;90(suppl 1):s2-s5.
  9. 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 ages 2 and up

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

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When an ERT is needed
When an ERT is needed

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Indication and Usage

Cerezyme® (imiglucerase) for injection is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions:

  • anemia
  • thrombocytopenia
  • bone disease
  • hepatomegaly or splenomegaly
Important Safety Information

Warnings and Precautions:

Hypersensitivity and Infusion-Associated Reactions:
Hypersensitivity reactions, some of which are serious and include anaphylaxis have been reported. Hypersensitivity and other infusion-associated reactions have been reported during or shortly after infusion and include pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, cough, cyanosis, tachycardia, and hypotension. Patients with antibody to imiglucerase have a higher risk of hypersensitivity reactions. Not all patients with symptoms of hypersensitivity have detectable IgG antibody. Consider periodic monitoring during the first year of treatment for IgG antibody formation.

If a severe hypersensitivity reaction occurs, discontinue Cerezyme treatment and initiate appropriate medical treatment. Consider the risks and benefits of readministering Cerezyme to individual patients following a severe reaction. If Cerezyme is readministered, consider reducing the rate of infusion, pretreat with antihistamines and/or corticosteroids, and monitor patients for new signs and symptoms of a severe hypersensitivity reaction.

Adverse Reactions:
Adverse reactions reported in adults include back pain, chills, dizziness, fatigue, headache, hypersensitivity reactions, nausea, pyrexia, and vomiting.

Adverse reactions reported in pediatric patients 2 years of age and older are similar to adults.

Immunogenicity:
Approximately 15% of patients treated and tested to date have developed IgG antibody to Cerezyme during the first year of therapy. Patients who developed IgG antibody did so largely within 6 months of treatment and rarely developed antibodies to Cerezyme after 12 months of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity. Patients with antibody to Cerezyme have higher risk of hypersensitivity reaction.

Please see Full Prescribing Information (PDF).

To report SUSPECTED ADVERSE REACTIONS, contact Sanofi Medical Information at 1-800-745-4447, Option 2.