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

20-Year Long-term Study

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

The 20-year ICGG Gaucher Registry analysis

Study description3

The 20-year International Collaborative Gaucher Group (ICGG) Gaucher Registry study by Weinreb et al is a retrospective, observational, single-arm study of patients with Gaucher disease type 1 who were treated with Cerezyme* for about 20 years. Read more...

Cerezyme demonstrated improvements in visceral, hematologic,
and certain bone parameters over 20 years3*

A RETROSPECTIVE, OBSERVATIONAL, SINGLE-ARM STUDY OF PATIENTS WITH GAUCHER DISEASE TYPE 1 WHO HAD DOSE AND CLINICAL DATA AROUND FIRST INFUSION OF CEREZYME* AND OVER THE COURSE OF ≈20 YEARS OF THERAPY

Results for nonsplenectomized patients

Improvements in visceral parameters3

-77% decrease in mean spleen volume
-77% mean spleen volume†
First infusion: 18.2 MN
20 years following first infusion: 4.2 MN (n=74)
-44% decrease in mean liver volume
-44% mean liver volume†
First infusion: 1.8 MN
20 years following first infusion: 1.0 MN (n=74)

Improvements in hematologic parameters3

+2.4 g/dl increase in mean hemoglobin level
+2.4 g/dL mean hemoglobin level
First infusion: 11.4 g/dL
20 years following first infusion: 13.8 g/dL
(n=299)
+84% increase in mean platelet count
+84% mean platelet count†
First infusion: 92 x 102/mm3
20 years following first infusion: 169 x 103/mm3
(n=293)

Decrease in certain bone parameters related to Gaucher disease type 13

-13% patients reporting bone pain
-13%
patients reporting bone pain†
First infusion: 56 patients reported bone pain
20 years following first infusion: 49 patients reported bone pain
(n=118)
-77% patients reporting bone crisis
-77%
patients reporting bone crisis†
First infusion: 17 patients reported bone crisis
20 years following first infusion: 17 patients reported bone crisis
(n=113)

Mean changes from baseline at 10 and 20 years, respectively, in splenectomized patients were: changes were observed in liver volume: 2.3 MN to 1.1 MN and 1.0 MN; hemoglobin: 11.7 g/dL to 13.3 g/dL and 13.4 g/dL; platelet count: 229.1 × 109/L to 288.1 × 109/L and 257.0 × 109/L; without bone crisis: 52.2% to 91.3% and 100%; without bone pain: 16.3% to 30.6% and 46.9%.

* The Cerezyme treatment group from the Gaucher Registry analyses represents patients with Gaucher disease type 1 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.
    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.
Cerezyme improved bone pain, bone crises, and bone mineral density
Efficacy in certain Gaucher-related bone parameters

Cerezyme effect on bone pain, bone crises, and bone mineral density

See results
An established ERT for children
An established ERT for children ages 2 and up

Long-term improvements shown in pediatric patients with Gaucher disease type 1

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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.