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

Gaucher Prevalence

Gaucher disease is a rare, genetic disorder that causes the accumulationof glucosylceramide, also known as glucocerebroside, (GL-1) in cells, resulting in progressive, multi-organ dysfunction.1 Gaucher disease type 1 can be effectively managed once diagnosed.2

Gaucher disease type 1 is more prevalent than you think

More than 90% of Gaucher disease patients are type 1.2 Although Gaucher disease type 1 is a rare genetic condition, it’s important to understand its occurrence in different patient populations.

Estimated
prevalence of Gaucher disease type 12-4

GD1 is pan-ethnic, and occurs in 1 in 40,000 in the general population

Pan-ethnic, and occurs in ≈1 in 40,000
in general population

GD1 is more common in people of Ashkenazi Jewish ancestry

More common in people of Ashkenazi
Jewish ancestry

90% American Jews are Ashkenazi

American Jews are Ashkenazi

  • In patients of Ashkenazi Jewish ancestry, the frequency of Gaucher disease has a higher incidence (≈1 in 850) than hematologic malignancies (≈1 in 2,500)2
  • In patients of Ashkenazi Jewish ancestry, ≈1 in 15 is a carrier5
  • Despite the high frequency of Gaucher disease among patients of Ashkenazi Jewish ancestry, Gaucher disease type 1 is pan-ethnic3
    References:
  1. Mistry PK, Sadan S, Yang R, et al. Consequences of diagnostic delays in Type 1 Gaucher disease: the need for greater awareness among hematologists–oncologists and an opportunity for early diagnosis and intervention. Am J Hematol. 2007;82(8):697-701.
  2. Mistry PK, Cappellini MD, Lukina E, et al. A reappraisal of Gaucher disease-diagnosis and disease management algorithms. Am J Hematol. 2011;86(1):110-115.
  3. Weinreb NJ. Pathophysiology, clinical features, and natural history of Gaucher disease. Clin Adv Hematol Oncol. 2012;10(6) Suppl 8:3-6.
  4. Memorial Sloan Kettering Cancer Center. More Ashkenazi Jews have gene defect that raises inherited breast cancer risk. The Oncologist News Bulletin. 1996;1:335.
  5. Mistry PK. Genetics and diagnosis of Gaucher disease. Clin Adv Hematol Oncol. 2012;10(6) Suppl 8:7-9.
  6. Stirnemann J, Belmatoug N, Camou F, et al. A review of Gaucher disease pathophysiology, clinical presentation and treatments. Int J Mol Sci. 2017;18(2). pii: E441.
  7. Lachmann RH, Grant IR, Halsall D, et al. Twin pairs showing discordance of phenotype in adult Gaucher’s disease. Q J Med. 2004;97(4):199-204.
  8. Kaplan P, Andersson HC, Kacena KA, Yee JD. The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis. Arch Pediatr Adolesc Med. 2006;160(6):603-608.
  9. Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, Rosenbloom BE, Scott CR, Wappner RS, Weinreb NJ, Zimran A. The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease. Arch Intern Med. 2000 Oct 9;160(18):2835-2843.
  10. Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol. 2004;41(4)(suppl 5):4-14.
  11. Cox TM. Gaucher disease: clinical profile and therapeutic developments. Biol Targets Ther. 2010;4:299-313.
  12. Deegan PB, Cox TM. Imiglucerase in the treatment of Gaucher disease: a history and perspective. Drug Des Devel Ther. 2012;6:81-106.
  13. Shayman JA. Glucosylceramide synthase inhibitor treatment of Type 1 Gaucher disease. Drugs Future. 2010;35(8):613-620.
Gaucher disease Type 1 is manageable once diagnosed
Gaucher disease type 1 is manageable once diagnosed

When an ERT is needed, choose the longest-approved therapy

Learn more
An established ERT for children
An established ERT for children ages 2 and up

Studied in the largest reported group of pediatric patients with Gaucher disease type 1

Explore pediatric data
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.