Cerezyme (Imiglucerase)

What it is and what it treats

Cerezyme (imiglucerase) is a recombinant enzyme replacement therapy indicated for long-term treatment of patients with a confirmed diagnosis of Type 1 or Type 3 Gaucher disease, a rare inherited lysosomal storage disorder caused by deficient activity of the enzyme glucocerebrosidase.

In Gaucher disease, glucocerebroside accumulates within macrophages, leading to hepatosplenomegaly, skeletal complications, anaemia and thrombocytopenia. Imiglucerase supplies an exogenous, functionally active form of the enzyme, which is taken up by macrophages and catalyses hydrolysis of accumulated substrate, addressing the underlying biochemical defect rather than only its symptoms.

The product has an extensive treatment history spanning several decades and remains a reference enzyme replacement therapy for Gaucher disease, alongside other approved alternatives such as velaglucerase alfa and taliglucerase alfa, which prescribers may consider depending on individual patient circumstances and antibody status.

Presentations and dosage forms

• Lyophilised powder for concentrate for solution for infusion, 400 units per vial
• Single-use vials intended for reconstitution and further dilution prior to intravenous administration
• No oral, subcutaneous or intramuscular presentations are authorised

How it is administered

The EU SmPC describes imiglucerase as being administered by intravenous infusion. Individualised dosing is determined by the treating physician based on the severity of disease manifestations, and doses have historically ranged from 2.5 U/kg three times weekly up to 60 U/kg every two weeks in published regimens, with adjustment according to clinical and biochemical response.

Reconstituted and diluted solution is infused over a period typically described as one to two hours, with the rate that may be adjusted based on tolerability. Premedication with antihistamines has been used in clinical practice to reduce the likelihood of infusion-associated reactions in sensitised patients.

Treatment is intended to be continued long term, with periodic monitoring of haematological parameters, organ volumes and skeletal status used to guide dose adjustments over time. Administration should take place in a setting equipped to manage infusion reactions.

Storage and handling

• Store at 2–8 °C under continuous refrigeration; do not freeze
• Keep vials in the original carton to protect from light
• Reconstituted and diluted solution should be used within the timeframe specified in the SmPC when stored at 2–8 °C
• Do not use beyond the expiry date printed on the packaging
• Cold-chain integrity must be maintained throughout transport and storage prior to administration

Side effects

The following adverse reaction profile is based on the approved EU SmPC and long-term post-marketing experience with imiglucerase.

Commonly reported

Serious effects requiring medical attention

Patients and caregivers should be advised to report suspected adverse reactions to their treating physician or the relevant national reporting scheme; infusion-associated reactions should be evaluated promptly.

Cerezyme compared with other Gaucher disease enzyme replacement therapies

Imiglucerase was the first widely adopted enzyme replacement therapy for Gaucher disease and carries a long clinical history. Alternative enzyme replacement therapies, including velaglucerase alfa and taliglucerase alfa, are approved in various jurisdictions and differ in production cell line and dosing schedule.

Choice between these therapies is a specialist clinical decision that accounts for prior treatment response, antibody status, availability and individual patient factors, rather than a straightforward substitution.

Availability and supply route

Marketing authorisation status for Cerezyme varies by country, and prescribing information, approved indications and pack presentations may differ between the EU, US and other regulated markets. Clinicians and procurement teams should confirm local registration status before planning treatment.

Where a product is not registered or locally available in sufficient supply, named-patient or specials import frameworks exist in many jurisdictions to allow supply of a foreign-registered medicine against a valid prescription and appropriate regulatory authorisation, subject to the patient's treating physician and local import controls.

Pharmacy Remedy is a licensed Turkish pharmaceutical wholesaler that supplies medicines through licensed distribution channels to hospitals, pharmacies and other authorised institutions; it does not sell medicines directly to the public and all supply is conditional on valid prescriptions and applicable import documentation.

Frequently asked questions

Primary sources

• EMA EPAR — Cerezyme
• FDA prescribing information — Cerezyme

Further reading and regulatory routes

• Which country Cerezyme is sourced from, and why prices differ
• How to verify a medicine is genuine
• Why medicines cost less in Türkiye
• WHO falsified medicine alerts
• Named patient supply
• UAE import guide
• For healthcare professionals
• All medicine monographs

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• Fabrazyme (agalsidase beta)
• Ilaris (canakinumab)