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

8-Year Pediatric Study

Choose Cerezyme: an established ERT for children ages 2 and up1

View Indication and Usage

Choose Cerezyme: an established ERT for children ages 2 and up1,7

The safety and effectiveness of Cerezyme have been established in pediatric patients 2 years of age and older1:

  • Evidence from adequate and well-controlled studies of Cerezyme and alglucerase in adults and pediatric patients 12 years of age and older
  • Additional data obtained from the medical literature and from postmarketing experience in pediatric patients as young as 2 years of age
  • The safety and effectiveness in patients younger than 2 years have not been established

Cerezyme* improved long-term visceral and hematologic manifestations in pediatric patients in an 8-year ICGG Gaucher Registry study7

Study description (Andersson et al)7

This observational study used data derived from the International Collaborative Gaucher Group (ICGG) Gaucher Registry submitted between 1991 and January 2006. Data were retrospectively analyzed for all patients (n=884) with Gaucher disease type 1 who had intact spleens and were receiving alglucerase (22.4%) or Cerezyme (77.6%).Read more...

Cerezyme significantly reduced spleen and liver volumes in children

Studied in the largest reported group of treated pediatric patients (n=884) with Gaucher disease type 1 around the world7

Cerezyme* reduced spleen and liver volumes in children7

-79% decrease in spleen volume
-45% decrease in liver volume
  • Approximately half of the treatment effect was achieved after ≈1 year of treatment
  • Volume reductions continued over the entire 8-year study period

Improvements were seen in children treated with Cerezyme* over 8 years

Cerezyme* improved hematologic parameters in children7

+2 g/dl increase in mean hemoglobin level
+74% increase in mean platelet count
  • Improvements in median normalized hemoglobin level were demonstrated during the first year of treatment
  • Median hematologic parameters increased to levels similar to those in the normal population after 8 years

0% of children on Cerezyme had anemia after 6 years

Cerezyme* effect on bone mineral density (BMD) over 12 years in children7

Cerezyme increased bone mineral density over 12 years in children

The DXA Z-scores for patients in the 95th, 75th, 50th, 25th, and 5th percentiles at first infusion were 1.22, 0.49, -0.35, -1.19, and -1.93, respectively. At 12 years, the DXA Z-scores for patients in the 95th, 75th, 50th, 25th, and 5th percentiles were 1.87, 1.13, 0.29, -0.55, and -1.29. N=127; BMD Z-score intercepts and slopes (change over time) were monitored for 12 years.

Timing of pediatric treatment should consider that most bone mineral is accrued in the first 2 decades of life and BMD peaks in the third decade

* The Cerezyme treatment group from the Gaucher Registry analyses represents pediatric patients with Gaucher disease type 1 who received either alglucerase or imiglucerase.
† Normalized hemoglobin levels were analyzed as grams per deciliter below the lower limit of the reference range, defined on the basis of age- and gender-adjusted normal values.
‡ 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.
Established long-term safety data
Evaluated safety

Cerezyme has demonstrated long-term safety

View 10-year data
When is an ERT 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.