Recombinant glucocerebrosidase Pregnancy: Available data on more than 500 pregnancies from the international Gaucher Disease registry, postmarketing reports, published observational studies and case reports have not identified a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. No animal reproduction studies have been conducted. A pregnancy exposure registry monitors outcomes in women exposed during pregnancy. Pregnancy may exacerbate existing Type 1 Gaucher disease.

Imiglucerase

Brand names: Cerezyme

Imiglucerase is a recombinant enzyme replacement therapy used for the long-term treatment of type 1 (and selected type 3) Gaucher disease.

Adult dose

Dose: 2.5 units/kg three times a week to 60 units/kg once every two weeks — titrated to disease severity and therapeutic goals (dose is expressed in UNITS/kg, not mg/kg)

Route: Intravenous infusion — each vial reconstituted with 10.2 mL Sterile Water for Injection to 40 units/mL, then diluted in 0.9% sodium chloride injection and given through a 0.2 micron in-line low protein-binding filter

Frequency: Depending on the regimen chosen, from three times a week to once every two weeks

NO UK SPC IN BUNDLE — dose taken from US labelling (CEREZYME, Genzyme); verify against the UK SPC. Ensure physicians knowledgeable in the management of patients with Gaucher disease direct therapy, and administer under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis, in a healthcare setting with cardiopulmonary resuscitation equipment available. INFUSION DURATION: for adults and paediatric patients weighing greater than 20 kg, infuse over 1 to 2 hours; for paediatric patients weighing 20 kg or less, infuse over 2 hours. DILUTION VOLUMES at a dose of 60 units/kg: 1.5 kg to <6 kg — 12 mL in a syringe for infusion; 6 kg to <13 kg — 26 mL in a syringe; 13 kg to <=20 kg — 100 mL infusion bag; >20 kg to <=100 kg — 200 mL infusion bag; >100 kg — 400 mL infusion bag. If prescribed at a dose lower than 60 units/kg, dilute to a final concentration between 6 units/mL and 30 units/mL; if the total volume is 26 mL or less, administer by syringe pump. For patients who have experienced hypersensitivity reactions, premedicate with antihistamines and/or corticosteroids. Discontinue immediately and treat if a severe hypersensitivity or severe infusion-associated reaction occurs; for mild or moderate reactions consider decreasing the infusion rate, temporarily stopping the infusion, and/or giving antihistamines, antipyretics and/or corticosteroids. PAEDIATRIC: safety and effectiveness for non-CNS manifestations of Type 1 and Type 3 Gaucher disease have been established in paediatric patients, but the label gives no separate paediatric units/kg regimen — only the infusion-duration and dilution rules above. paedDose is left null because the dose is expressed in units/kg rather than mg/kg; verify the paediatric regimen against a children's formulary.

Dose independently re-sourced from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — clinician-reviewed. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

None stated (US label §4 states: None)

Side effects

Hypersensitivity reactions including anaphylaxis — pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnoea, cough, cyanosis, tachycardia and hypotension
Infusion-associated reactions
Headache and dizziness
Back pain
Chills, pyrexia and fatigue
Nausea and vomiting

Clinical monograph

How it works

It substitutes for deficient glucocerebrosidase, hydrolysing accumulated glucocerebroside in macrophages and reducing organomegaly and haematological complications.

Prescribing in practice

Hypersensitivity and infusion-related reactions can occur and may be managed by slowing the infusion, pre-medication or antihistamines as needed.
It should be prescribed and supervised by a specialist in Gaucher disease or lysosomal storage disorders.
Antibody formation can develop; monitor patients who show reduced response or reactions per current prescribing references.

Monitoring

Monitor haematological parameters, organ volumes and biomarkers of disease activity to assess and adjust therapy.

Counselling the patient

Infusions are given on a regular schedule with monitoring for reactions.
Report rash, flushing, breathlessness or itching during or after an infusion.
Continued treatment is needed to maintain the benefit on blood counts and organ size.

Evidence & guidelines

Enzyme replacement is the established standard of care for type 1 Gaucher disease, supported by long-term registry and trial data.

Reference: NICE highly specialised technology; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

Drugs

Velaglucerase alfa · Recombinant glucocerebrosidase

Pathways