The ONLY ERT (enzyme replacement therapy) with over 30 years of real-world experience treating Gaucher disease type 1. The first and only treatment approved for non-CNS manifestations Gaucher disease type 3.1,2
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.1,3-7 The long-term safety of Cerezyme was shown in an international monitoring database.8
Explore the findings of several 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 or type 3.1,3,4-7
Visceral &
Hematologic Data
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Parameters assessed: Visceral and hematologic |
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2-Year, Long-Term Registry Study1 Parameters assessed: Visceral, hematologic, and pediatric growth |
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20-Year, Long-Term Registry Study3,a Parameters assessed: Visceral, hematologic, bone pain, and bone crises |
Bone Data
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48-Month, Long-Term Prospective Study4 Parameters assessed: Bone mineral density (BMD), bone pain, and bone crises |
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4-year, Long-Term Registry Study5 Parameters assessed: Bone pain and bone crises |
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8-Year, Long-Term Registry Study6 Parameters assessed: BMD |
Pediatric Data
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8-Year, Long-Term Registry Study7,a Parameters assessed: Visceral, hematologic, and BMD |
aThe Cerezyme treatment group from these 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 experts in the management of Gaucher disease.9
- The ICGG Gaucher Registry provides observational data on >7000 patients with Gaucher disease worldwide10
Indication
References: 1. Cerezyme (imiglucerase). Prescribing information. Genzyme Corporation, Cambridge, MA. 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 Jr, 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. 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. 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. 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. 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. 9. National Gaucher Foundation. Should your medical history be in the International Gaucher Registry? Accessed November 17, 2025. https://www.gaucherdisease.org/blog/medical-history-international-gaucher-registry/ 10. Mistry PK, Balwani M, Charrow J, et al. Long-term effectiveness of eliglustat treatment: a real-world analysis from the International Collaborative Gaucher Group Gaucher Registry. Am J Hematol. 2024;99(8):1500-1510.
