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Cerezyme® has demonstrated long-term efficacy, and established safety profiles with 20 years of real-world experience in the treatment of Gaucher disease1–4

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Demonstrated efficacy in visceral and haematological parameters5,6

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Demonstrated efficacy in certain bone parameters7–10

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Demonstrated efficacy in children treated globally*11–14

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Individualised dosing for optimal response1

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Established safety profile1,3,4

*The Cerezyme® treatment group from the Gaucher Registry studies represents patients who received either Ceredase (alglucerase) or Cerezyme® initially and continued treatment with Cerezyme® long term.4,5,11,12,14,15


The Gaucher Registry is the world’s largest cooperative observational database for Gaucher disease, providing data on more than 6,000 patients worldwide.16 Data from the Gaucher Registry is used to assess the effectiveness of treatment for symptoms commonly experienced by patients with Gaucher disease such as splenomegaly, thrombocytopenia, hepatomegaly and bone pain.3,4,9,12,16

Visceral and haematological parameters

Cerezyme® has shown both early and long-term improvements in visceral and haematological parameters5,6

Efficacy - bone health

Skeletal complications of Gaucher disease are progressive and therefore, early initiation of Cerezyme® could improve bone outcomes for patients10

Bone involvement is the principal cause of pain, disability and reduced quality of life in Gaucher disease patients of all ages.17 The pathophysiology responsible for bone involvement in Gaucher disease is not completely understood.17

Efficacy - paediatric patients

Cerezyme® improved visceral, haematological, certain bone parameters, and normalised growth and puberty vs. baseline11–14

Published paediatric data for type 1 patients from the Gaucher Registry demonstrated:

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Cerezyme® significantly reduced spleen and liver volumes in children vs. baseline11

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Cerezyme® significantly improved haematological parameters in children vs. baseline11

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Cerezyme® can normalise growth in children with Gaucher disease and result in catch-up growth during treatment (relative to pre-treatment height)11

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Cerezyme® increased BMD in the lumbar spine vs. baseline11

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Cerezyme® reduced or eliminated bone crisis in children during the study duration, compared to before treatment initiation11

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Cerezyme® resulted in effective amelioration of osteopenia in children and adolescents vs. baseline12

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Cerezyme® reduced or eliminated bone pain and improved bone lesions in children within 26 months vs. baseline13

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Cerezyme® normalised growth and time of puberty onset in most patients14

In bone and growth studies, the Cerezyme® treatment group from the Gaucher Registry studies represents patients who received either alglucerase or imiglucerase initially and continued treatment with imiglucerase long term.5,10–12,15

Dose and safety

Cerezyme® administration and safety information

Cerezyme® treatment should be individualised for each patient and disease management should be directed by physicians knowledgeable in the treatment of Gaucher disease.1

Selecting dosage1

Initial doses of 60 U/kg of body weight once every 2 weeks have shown improvement in haematological and visceral parameters within 6 months of therapy and continued use has either stopped progression of or improved bone disease. Administration of doses as low as 15 U/kg of body weight once every 2 weeks has been shown to improve haematological parameters and organomegaly, but not bone parameters.

No dose adjustment is necessary for the paediatric population.

Selecting dosage


At initial infusions, Cerezyme® should be administered at a rate not exceeding 0.5 unit per kg body weight per minute, i.e. 120 min for a patient whose dose is 60 U/kg, whatever the patient’s weight.

Maximum infusion rate is 1 unit per kg body weight per minute, i.e. 60 min for a patient whose dose is 60 U/kg, whatever the patient’s weight.

Infusions should be administered every 2 weeks.


Treatment location1

Initially treatment should be administered in a clinical setting. Infusion of Cerezyme® at home may be considered for patients who are tolerating their infusions well for several months. Self-infusion at home also requires training of the patient or caregiver by a healthcare professional in a clinical setting.

Initial infusion rate should not exceed 0.5 unit/kg/min. At subsequent infusions, infusion rate may be increased. Patients experiencing adverse events during the infusion need to immediately stop the infusion process and seek the attention of a healthcare professional.

Treatment location

Adverse reactions are listed by system organ class and frequency in the table below. Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.1

MedDRA system organ class Common
(≥1/100 to <1/10)
(≥1/1,000 to <1/100)
(≥1/10,000 to <1/1,000)
Nervous system disorders Dizziness, headache, paraesthesia*
Cardiac disorders Tachycardia*, cyanosis*
Vascular disorders Flushing*, hypotension*
Respiratory, thoracic and mediastinal disorders Dyspnoea*, coughing*
Gastrointestinal disorders Vomiting, nausea, abdominal cramping, diarrhoea
Immune system disorders Hypersensitivity reactions Anaphylactoid reactions
Skin and subcutaneous tissue disorders Urticaria/angioedema*, pruritus*, rash*
Musculoskeletal and connective tissue disorders Arthralgia, backache*
General disorders and administration site conditions Infusion site discomfort, infusion site burning, infusion site swelling, injection site sterile abscess, chest discomfort*, fever, rigors, fatigue

Symptoms suggestive of hypersensitivity (* marked in the table above) have been noted, overall in approximately 3% of the patients. Onset of such symptoms has occurred during or shortly after infusions. These symptoms generally respond to treatment with antihistamines and/or corticosteroids. Patients should be advised to discontinue infusion of the product and contact their physician if these symptoms occur.

During the first year of treatment, immunoglobulin G antibodies to Cerezyme® may form in approximately 15% of patients. Patients with antibodies to Cerezyme® have a higher risk of hypersensitivity reactions.

Limited experience from 150 pregnancy outcomes suggests that Cerezyme® is beneficial in controlling the underlying Gaucher disease during pregnancy. Risk-benefit treatment assessment is required for each pregnancy. Consult section 4.6 of SmPC.

Cerezyme® mechanism of action

Cerezyme® is a modified form of the enzyme β-glucocerebrosidase which reduces the accumulation of glucosylceramide (GL-1). Cerezyme® is an enzyme replacement therapy (ERT) with demonstrated long-term efficacy, an established safety profile and real-world experience in the treatment of Gaucher disease.1,3,4

Cerezyme® reduces the build-up of GL-1 because the modified form of the enzyme, β-glucocerebrosidase, breaks down GL-1 into glucose and ceramide. By replacing the missing enzyme, Cerezyme® can reduce the accumulation of GL-1 in patients with non-neuronopathic (type 1) or chronic neuronopathic (type 3) Gaucher disease who exhibit clinically significant non-neurological manifestations of the disease.1

Since 1991, The Gaucher Registry has collected voluntary information from over 6,000 people internationally.2 Data from the Registry sponsored by Sanofi Genzyme is used to help researchers and physicians understand the impact of Gaucher disease and the effectiveness of long-term treatment.

Useful links


DDI Tool

This tool has been designed to help quickly navigate through the drug-drug interactions (DDIs) for Cerdelga® as outlined in the SmPC based on the metabolizer status of a patient.
Prescribing decisions should be made in consultation with an expert in the management of patients with Gaucher disease.



UK-based charity dedicated to raising awareness of Gaucher disease, and those affected and their families. Provides support and information for those affected by Gaucher disease.



To support you in identifying and treating your patients with Gaucher disease, Sanofi Genzyme have launched a free access e-learning module: Identifying and Treating Gaucher disease.


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