Cerezyme 400 U powder for concentrate for solution for infusion – imiglucerase for type 1 and type 3 Gaucher disease

Cerezyme contains imiglucerase – a man-made (recombinant) form of the enzyme acid beta-glucosidase, which is missing or not active enough in people with Gaucher disease. In this rare inherited metabolic disease, glucosylceramide, a substance made of sugar and fat, builds up in the body.

The medicine is intended for long-term enzyme replacement therapy and is given as an intravenous infusion under the supervision of a doctor experienced in treating Gaucher disease. In Serbia it is authorised as a 400-unit powder for concentrate for solution for infusion (manufacturer Genzyme Ireland Limited, marketing authorisation holder Amicus SRB d.o.o.).

What is Cerezyme used for and how is it given?

Key facts about the medicine

What is Cerezyme used for?

Cerezyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of non-neuronopathic (type 1) or chronic neuronopathic (type 3) Gaucher disease who have clinically significant non-neurological manifestations of the disease:

• anaemia (after exclusion of other causes, e.g. iron deficiency);
• thrombocytopenia (low platelet count, tendency to bleed);
• bone disease (after exclusion of other causes, e.g. vitamin D deficiency);
• enlarged liver or spleen.

The leaflet applies to all ages – children, adolescents, adults and elderly patients. The effect of the medicine on neurological symptoms of Gaucher disease has not been established.

How is it used and dosed?

• The dose is set individually according to the severity of symptoms and other factors. The recommended dosage in the leaflet is 60 units/kg body weight once every two weeks.
• According to the SmPC, lower doses (e.g. 15 U/kg every other week) also improve blood counts and organ size, but not bone changes; the doctor may reduce or increase the dose.
• The medicine is prepared by mixing the powder with sterile water, then diluted in 0.9% sodium chloride solution and given as an infusion into a vein.
• Infusion rate: at first infusions no more than 0.5 U/kg/min, later no more than 1.0 U/kg/min – rate increases only under the supervision of a healthcare professional.
• The doctor monitors the response to treatment every 6 to 12 months.
• The medicine must not be mixed with other medicines in the same infusion.

This is the general dosage from the leaflet and SmPC – your dose and infusion schedule are determined by your doctor.

Special patient groups

• Children and adolescents: no dose adjustment needed; the leaflet applies to all ages.
• Elderly patients: the leaflet also applies to elderly patients.
• Chronic neuronopathic Gaucher disease: there are no data on the effect on central nervous system symptoms, so no special dosing regimen can be recommended for these symptoms.
• Sodium-controlled diet: one vial contains 41 mg sodium (2% of the recommended maximum daily intake), and the medicine is given in sodium chloride solution.

Contraindications – when the medicine must not be used

• allergy (hypersensitivity) to imiglucerase or any of the excipients

Warnings and precautions

• Allergic reactions may occur during or shortly after the infusion – your doctor may test whether you are allergic to imiglucerase. According to the SmPC, about 15% of patients develop IgG antibodies to the medicine during the first year, and their risk of hypersensitivity reactions is higher.
• In patients who previously had antibodies or hypersensitivity to alglucerase (Ceredase), the medicine is used with caution.
• Report shortness of breath to your doctor – pulmonary hypertension is a known complication of Gaucher disease, regardless of treatment.
• The medicine contains 41 mg sodium per vial.

Interactions with other medicines, food and drink

• No interaction studies have been performed (SmPC).
• The medicine must not be given mixed with other medicines in the same infusion.
• Tell your doctor about all medicines you are taking.

Pregnancy and breast-feeding

Talk to your doctor if you are pregnant, breast-feeding or planning a pregnancy; the leaflet recommends caution. According to the SmPC, limited experience (about 150 pregnancies) shows that the medicine is beneficial in controlling Gaucher disease during pregnancy; in women already receiving it, continuing treatment should be considered, with careful monitoring and individual dosing. It is not known whether the medicine passes into breast milk, but the enzyme is likely to be digested in the baby’s gut.

Driving and using machines

According to the SmPC, Cerezyme has no or negligible influence on the ability to drive and use machines.

Possible side effects

• Common: shortness of breath, cough, hives (swelling of the skin or lining of the mouth and throat), itching, skin rash.
• Uncommon: dizziness, headache, tingling or numbness of the skin, fast heartbeat, bluish skin, flushing, drop in blood pressure, vomiting, nausea, abdominal cramps, diarrhoea, joint pain, discomfort, burning, swelling or sterile abscess at the infusion site, chest discomfort, fever, rigors, tiredness, back pain.
• Rare: anaphylactoid reaction.
• Most reactions occur during or shortly after the infusion; the doctor may give antihistamines and/or corticosteroids to prevent an allergic reaction.

Report suspected side effects to your doctor, pharmacist, nurse or ALIMS.

Overdose, missed dose and stopping treatment

• Overdose: no cases have been reported; according to the SmPC doses up to 240 U/kg every other week have been used.
• Missed infusion: contact your doctor.
• Stopping treatment: any change or interruption is decided by the doctor managing your treatment.

Related information

The official texts of the patient leaflet and the summary of product characteristics are available on the ALIMS website (links in the Sources section). For questions about treatment and home infusion, contact the centre where you are being treated.

Composition, pack and manufacturer

• Active substance: imiglucerase, 400 units per vial (40 U/mL after reconstitution); produced by recombinant DNA technology.
• Excipients: mannitol; sodium citrate; citric acid monohydrate; polysorbate 80.
• Pack: 20 mL type I borosilicate glass vial with siliconised butyl stopper and flip-off cap; the vial contains a 0.6 mL overfill for accurate dosing; 1 vial per carton.
• Marketing authorisation holder: AMICUS SRB D.O.O., Milorada Jovanovića 9, Belgrade. Manufacturer: Genzyme Ireland Limited, Ireland.

Storage

• Store unopened vials in a refrigerator (2–8 °C).
• The reconstituted solution is not stored – it is diluted immediately; the diluted solution can be stored for up to 24 hours at 2–8 °C, protected from light.
• Shelf life of the unopened vial: 3 years.
• Keep out of the sight and reach of children.

Frequently asked questions

How often is Cerezyme given?

Usually once every two weeks as an infusion into a vein. The dose (recommended 60 U/kg) and frequency are set individually.

Can I have infusions at home?

Yes, if your doctor considers that you meet the conditions – usually when previous infusions over several months were well tolerated, and after training in hospital.

Does Cerezyme treat the neurological symptoms of Gaucher disease?

No – the effect on neurological symptoms has not been established. The medicine is intended for non-neurological manifestations: anaemia, thrombocytopenia, bone disease and enlargement of the liver and spleen.

What if I miss an infusion?

Contact the doctor managing your treatment to arrange the next infusion.

Sources

• ALIMS – Summary of Product Characteristics Cerezyme®, 400 U, powder for concentrate for solution for infusion (Serbian)
• ALIMS – Patient leaflet Cerezyme®, 400 U, powder for concentrate for solution for infusion (January 2023, Serbian)

Sources checked on: 24 October 2026.