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

10-Year Long-Term Study

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

The 10-year ICGG Registry analysis

Study description3

The 10-year ICGG Gaucher Registry study by Weinreb et al is a retrospective, observational, single-arm study of patients with Gaucher disease type 1 who had dose and clinical data at first infusion of Cerezyme* and after 10 years of therapy. Read more...

Cerezyme demonstrated statistically significant improvements in visceral, hematologic,
and certain bone parameters over 10 years3*

Improvements in visceral parameters3

-73% decrease in mean spleen volume
-73% mean spleen volume
First infusion: 19.4 MN 10 years following first infusion: 5.2 MN (P<0.0001; n=107)
-44% decrease in mean liver volume
-44% mean liver volume
First infusion: 1.8 MN 10 years following first infusion: 1.0 MN (P<0.0001; n=105)

Improvements in hematologic parameters3

+2.4 g/dl increase in mean hemoglobin level
+2.4 g/dL
mean hemoglobin level

First infusion: 11.2 g/dL 10 years following first infusion: 13.6 g/dL
(P<0.0001; n=376)
+75% increase in mean platelet count
+75% mean platelet count
First infusion: 95.3 x 102/mm3 10 years following first infusion: 166.66 x 103/mm3
(P<0.0001; n=397)

Decrease in certain Gaucher-related bone parameters3

-57% patients reporting bone pain
-57%
patients reporting bone pain

First infusion: 98/187 patients reported bone pain 10 years following first infusion: 42/187 patients reported bone pain
(P<0.0001; n=98)
-93% patients reporting bone crisis
-93%
patients reporting bone crisis

First infusion: 27/169 patients reported bone crisis 10 years following first infusion: 2/169 patients reported bone crisis
(P<0.0001; n=27)

Data for the nonsplenectomized patient group are shown above.
*In a retrospective, observational, single-arm study of patients with Gaucher disease type 1 who had bone and clinical data at first infusion of Cerezyme and after 10 years of therapy. 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.
Cerezyme improved bone pain, bone crises, and bone mineral density
Efficacy in certain Gaucher-related bone parameters

Cerezyme improved bone pain, bone crises, and bone mineral density

See results
An established ERT for children
An established ERT for children

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

Explore pediatric data
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).