Fabrazyme 35 mg – agalsidase beta enzyme replacement therapy for Fabry disease

Fabrazyme contains agalsidase beta, an enzyme used as enzyme replacement therapy in Fabry disease – an inherited disease in which the enzyme α-galactosidase is absent or less active than normal. As a result, a fatty substance called globotriaosylceramide (GL-3) is not cleared from cells and builds up in the walls of the blood vessels of organs.

According to the leaflet, Fabrazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease – adults, children from 8 years, adolescents and the elderly. It is given by intravenous infusion, only under the supervision of a specialist experienced in treating this disease.

What is Fabrazyme used for and how is it given?

Key facts about the medicine

What is Fabrazyme used for?

Long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease – in adults, children aged 8 years and over, adolescents and the elderly.

How is it given and dosed?

• The medicine is given by intravenous infusion (drip); the powder is reconstituted with water for injections and diluted with 0.9% sodium chloride solution before use – prepared by a healthcare professional.
• Adults: 1 mg/kg body weight once every 2 weeks.
• Children aged 8 to 16 years: 1 mg/kg body weight once every 2 weeks.
• No dose adjustment is needed in kidney disease.
• The initial infusion rate must not exceed 0.25 mg/min (15 mg/hour); later it may be increased gradually if the infusion is well tolerated (in patients under 30 kg the rate stays at a maximum of 0.25 mg/min).
• Your doctor may allow home treatment if certain criteria are met.

Special patient groups

• Children 0–4 years: no clinical studies have been performed.
• Children 5–7 years: risks and benefits have not been established, so no dose can be recommended for this age.
• Kidney disease: the dose is not changed.

Contraindications – when the medicine must not be used

You must not receive Fabrazyme if you are allergic (hypersensitive) to agalsidase beta or any of the excipients.

Warnings and precautions

During treatment, infusion-associated reactions may occur – side effects during the infusion or until the end of the day of the infusion. If this happens, tell your doctor immediately; an additional medicine may be needed to prevent such reactions. The medicine contains less than 1 mmol (23 mg) sodium per vial.

Interactions with other medicines, food and drink

Tell your doctor about all medicines you take, especially any medicine containing chloroquine, amiodarone, benoquin or gentamicin – there is a theoretical risk that they reduce agalsidase beta activity.

Pregnancy and breast-feeding

Experience in pregnancy is limited; as a precaution, use during pregnancy should be avoided. Fabrazyme passes into breast milk – discuss with your doctor the benefits of breast-feeding versus continuing treatment. No studies on fertility have been performed.

Driving and using machines

Do not drive or use machines if you feel faint, drowsy or dizzy during or shortly after administration – talk to your doctor first.

Possible side effects

Side effects occur mostly during or shortly after the infusion. Severe, life-threatening allergic (anaphylactoid) reactions have been reported in a few patients.

• Very common: chills, fever, feeling cold, nausea, vomiting, headache, tingling and pins and needles.
• Common: chest pain, difficulty breathing, fast or slow heartbeat, palpitations, flushing and feeling hot, itching, rash, hives, swelling of the face or throat, throat tightness, dizziness, drowsiness, tiredness, abdominal, muscle, joint and back pain, stuffy nose, cough, wheezing, high or low blood pressure, fainting, ringing in the ears and others.
• Uncommon: bronchospasm, difficulty swallowing, infusion-site reactions and pain, flu-like illness, skin discolouration, cold extremities and others.
• Frequency not known: lower blood oxygen levels, serious inflammation of blood vessels.

Your doctor may slow the infusion or give additional medicines to prevent reactions. In patients whose dose was reduced long-term, some symptoms of Fabry disease were reported more often.

Overdose, missed dose and stopping treatment

• Overdose: doses up to 3 mg/kg body weight have been shown to be safe.
• Missed infusion: tell your doctor.
• Stopping treatment: any dose change or stopping is decided by the specialist.

Related information

Official information on the medicine is in the package leaflet and Summary of Product Characteristics on the ALIMS website (links in the “Sources” section).

Composition, pack and manufacturer

• Active substance: agalsidase beta – 35 mg per vial.
• Excipients: mannitol; sodium dihydrogen phosphate monohydrate; disodium hydrogen phosphate heptahydrate; nitrogen.
• Appearance: white to off-white powder; after reconstitution a clear, colourless liquid that must be further diluted.
• Pack: clear glass vial (type I) of 20 mL with siliconised butyl stopper and aluminium cap; carton with 1 vial.
• Marketing authorisation holder: Amicus SRB d.o.o., Milorada Jovanovića 9, Belgrade.

Storage

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

Frequently asked questions

How often is Fabrazyme given?

The recommended dose for adults and children aged 8 to 16 is 1 mg/kg body weight once every 2 weeks, by intravenous infusion.

What are infusion-associated reactions?

Side effects occurring during the infusion or until the end of that day – most often chills, fever, feeling cold, nausea, vomiting and headache. Your doctor may slow the infusion or give a medicine to prevent them.

Can Fabrazyme be given at home?

According to the leaflet, your doctor may advise home treatment if you meet certain criteria; the dispensing status exceptionally allows prescription supply to continue therapy at home.

Does the dose need changing in kidney disease?

No. According to the leaflet, no dose adjustment is needed in patients with kidney disease.

Sources

• ALIMS – Summary of Product Characteristics, Fabrazyme® 35 mg powder for concentrate for solution for infusion (SmPC, Serbian)
• ALIMS – Package leaflet, Fabrazyme® 35 mg (PIL, Serbian)

Sources checked on: 25 October 2026.