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Cerezyme® (imiglucerase) Studies Overview


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 years1-3

Cerezyme efficacy & safety studies overview

Cerezyme was proven to be safe and effective in 2 prospective clinical trials and was evaluated in multiple studies from the International Collaborative Gaucher Group (ICGG) Gaucher Registry. The long-term safety of Cerezyme was shown in an international monitoring database.3-7

Explore the findings of 6 studies evaluating the safety and efficacy of Cerezyme in visceral, hematologic, and certain bone parameters in adult and pediatric populations with Gaucher disease type 1.1,3-7

Visceral &
Hematologic Data

6-Month Pivotal Trial1
 

Parameters assessed: Visceral and hematologic parameters

20-Year, Long-Term Registry Study3,a

Parameters assessed: Visceral, hematologic, bone pain, and bone crises

Bone Data

48-Month, Long-Term Prospective Study4

Parameters assessed: Bone mineral density (BMD) and bone crises

4-year, Long-Term Registry Study6

Parameters assessed: Bone pain and bone crises

8-Year, Long-Term Registry Study5

Parameters assessed: BMD

Pediatric Data

8-Year, Long-Term Registry Study7,a
 

Parameters assessed: Visceral, hematologic, and BMD

aThe Cerezyme treatment group from the ICGG Gaucher Registry analyses represents patients with Gaucher disease type 1 who received either alglucerase or imiglucerase.

The International Collaborative Gaucher Group (ICGG) Gaucher Registry

The ICGG Gaucher Registry is the world’s largest cooperative, observational database on Gaucher disease. The ICGG Gaucher Registry is sponsored by Sanofi and directed by the ICGG, a group of physicians who are experts in the management of Gaucher disease.8

  • The ICGG Gaucher Registry provides observational data on more than 7000 patients with Gaucher disease worldwide3

Choose the longest-studied ERT

Supported by 10-year safety data from an international monitoring database

See how Cerezyme works

Designed to reduce accumulation of the lipid GL-1

Indication

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

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis, and to seek immediate medical care should symptoms occur.

Warnings and Precautions:

Hypersensitivity Reactions Including Anaphylaxis: See Boxed WARNING.
Patients with antibody to imiglucerase have a higher risk of hypersensitivity reactions. Consider periodic monitoring during the first year of treatment for lgG antibody formation.

Consider risks and benefits of readministering Cerezyme to individual patients following a severe reaction. 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.

Infusion-Associated Reactions:
Infusion associated reactions (IARs) have been observed in patients treated with Cerezyme. If an IAR occurs, decreasing the infusion rate, temporarily stopping the infusion and/or administering antihistamines and/or antipyretics may ameliorate the symptoms. Closely monitor patients who have experienced IARs when re-administering Cerezyme.

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.

Please see full Prescribing Information, including Boxed WARNING.

Indication

Important Safety Information

References: 1. Cerezyme (imiglucerase). Prescribing information. Genzyme Corporation, Cambridge, MA. 2. Weinreb N, Taylor J, Cox T, Yee J, vom Dahl S. Am J Hematol. 2008;83(12):890-895. 3. Weinreb NJ, Camelo JS, Charrow J, et al. Mol Genet Metab. 2021;123(2):100-111. 4. Sims KB, Pastores GM, Weinreb NJ, et al. Clin Genet. 2008;73(5):430-440. 5. Wenstrup RJ, Kacena KA, Kaplan P, et al. J Bone Miner Res. 2007;22(1):119-126. 6. Charrow J, Dulisse B, Grabowski GA, Weinreb J. Clin Genet. 2007;71(3):205-211. 7. Andersson H, Kaplan P, Kacena K, Yee J. Pediatrics. 2008;122(6):1182-1190. 8. Weinreb NJ, Kaplan P. Am J Hematol. 2015;90(suppl 1):s2-s5.

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