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

8-Year Pediatric Study

Choose Cerezyme: an established ERT for children1

View Indications and Usage

Choose Cerezyme: an established ERT for children1

The safety and effectiveness of Cerezyme have been established in patients between 2 and 16 years of age1:

  • Evidence from adequate and well-controlled studies of Cerezyme and alglucerase in adults and pediatric patients
  • Additional data obtained from the medical literature and from long-term postmarketing experience
  • Cerezyme has been administered to patients younger than 2 years of age; however the safety and effectiveness in patients younger than 2 years have not been established

Cerezyme* significantly improved long-term visceral and hematologic manifestations in pediatric patients in an 8-year ICGG Gaucher Registry study8

Study description (Andersson et al)8

This observational study used data derived from the 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 world8

Cerezyme* significantly reduced spleen and liver volumes in children8

-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* for 8 years

Cerezyme* significantly improved hematologic parameters in children8

+2 g/dl increase in mean hemoglobin level
+74% increase in mean platelet count
  • Significant 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* increased bone mineral density (BMD) over 12 years in children8

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 and had P values of 0.047 and 0.069, respectively.

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 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.

    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.
Established long-term safety data
Established safety

Cerezyme has demonstrated long-term safety

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When is an ERT 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).