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Agalsidase beta parenteral

Updated 2 Feb 2023 | Fabry's disease


Parenteral formulations of agalsidase beta.

Agalsidase beta is a recombinant form of alpha-galactosidase A and is produced by recombinant DNA technology using a mammalian Chinese Hamster Ovary (CHO) cell culture. The amino acid sequence of the recombinant enzyme and the nucleotide sequence that encoded it, are identical to the natural form.

Drugs List

  • agalsidase beta 35mg powder for solution for infusion
  • agalsidase beta 5mg powder for solution for infusion
  • FABRAZYME 35mg powder for concentrate for solution for infusion
  • FABRAZYME 5mg powder for concentrate for solution for infusion
  • Therapeutic Indications


    Enzyme replacement therapy - Fabry disease


    Treatment should be directed by a physician experienced in the treatment of Fabry disease.

    Infusion of agalsidase beta at home may be considered for patients who are tolerating their infusions well, after evaluation and recommendation by the treating physician.
    Dose and infusion rate should remain constant while at home, and not being changed without supervision of a healthcare professional.

    Patients experiencing adverse events during the home infusion need to immediately stop the infusion process and seek medical attention. Subsequent infusion may need to occur in a clinical setting.


    The recommended dose is 1mg/kg body weight administered once every 2 weeks.


    Safety and efficacy in patients over 65 years of age have not been established. No dosage regimen can be recommended in these patients at present.


    Children aged 8 years and older
    The recommended dose is 1mg/kg body weight administered once every 2 weeks.


    For intravenous infusion after reconstitution and dilution.

    The initial infusion rate should be not more than 0.25mg/min (15mg/hour). After patient tolerance is well established, the infusion rate may be increased gradually with subsequent infusions.

    The manufacturer recommends that the diluted solution be administered through an in-line low protein-binding 0.2 micron filter to remove any protein particles which will not result in a loss of agalsidase beta activity.


    Children under 8 years

    Precautions and Warnings

    Antibodies to agalsidase beta
    Patients over 65 years

    Advise ability to drive/operate machinery may be affected by side effects
    Consider pre-medication with antihistamines and/or antipyretics
    Treatment to be initiated and supervised by a specialist
    Dilute and use as an infusion
    If adverse reactions occur, reduce rate or temporarily stop infusion
    Record name and batch number of administered product
    Monitor periodically for IgG antibody formation
    Discontinue if serious allergic or anaphylactic reaction occurs

    It is expected that the majority of patients will develop IgG antibodies to agalsidase beta, typically within three months of the first infusion. Patients with IgG antibodies have a higher risk of infusion-associated hypersensitivity reactions. Patients who have developed infusion-associated reactions should be treated with caution when re-administering the product.

    Allergic-type hypersensitivity reactions are possible, as with all intra-venous protein products. Discontinue the infusion immediately in the event of a severe allergic or anaphylactic-type reaction and initiate appropriate treatment in accordance with the current medical standards for emergency treatment. Careful rechallenge of agalsidase beta can be performed at a low dose and low infusion rate. Once the patient tolerates infusion, the dose may be increased to reach therapeutic dose of 1mg/kg and the infusion rate may be increased by slowly titrating upwards as tolerated.

    The effect of agalsidase beta treatment on the kidneys of patients with advanced renal disease may be limited.

    Pregnancy and Lactation


    Use agalsidase beta with caution during pregnancy.

    The manufacturer recommends that agalsidase beta should not be used unless clearly necessary. Reproduction studies in rats have not shown a relation to agalsidase beta and impaired fertility or foetal harm. It is unknown whether agalsidase beta crosses the human placenta, however it's large molecular weight and short elimination half-life suggest that the enzyme may not cross to the foetus. At the time of writing there is no data on the use of agalsidase beta during human pregnancy, however, there has been one case report on the use of agalsidase alfa during pregnancy, resulting in the birth of a healthy male foetus. Briggs (2015), concludes that the animal data on agalsidase beta suggests that it is low risk when used in pregnancy, but the lack of human data prevents a complete foetal risk assessment. Due to the nature of Fabry disease, Briggs (2015) advises that if a pregnant woman requires enzyme replacement therapy and gives consent, agalsidase beta should not be withheld.


    Use agalsidase beta with caution during breastfeeding.

    The manufacturer recommends to stop breastfeeding when agalsidase beta is used as there are no data available on the effects in neonates exposed to agalsidase beta via breast milk. It is not known whether agalsidase beta is excreted in human milk, however, Briggs (2015) concludes that the large molecular weight and short half-life suggest that agalsidase beta may not be excreted into breast milk, and if excreted, may be digested by the nursing infant's gut.

    Side Effects

    Abdominal discomfort
    Abdominal pain
    Anaphylactic reaction
    Angioneurotic oedema
    Antibody formation
    Auricular swelling
    Back pain
    Burning sensation
    Chest discomfort
    Chest pain
    Ear pain
    Erythematous rash
    Extremity pain
    Facial oedema
    Facial swelling
    Hot flushes
    Hypersensitivity reactions
    Increased lacrimation
    Influenza-like symptoms
    Infusion-related symptoms
    Injection site reactions
    Leukocytoclastic vasculitis
    Livedo reticularis
    Local pain (injection site)
    Maculopapular rash
    Muscle spasm
    Muscle tension
    Musculoskeletal pain
    Nasal congestion
    Ocular hyperaemia
    Ocular pruritus
    Oral hypoaesthesia
    Oxygen saturation decreased
    Peripheral coldness
    Peripheral oedema
    Pharyngolaryngeal pain
    Sensation of cold
    Sensation of heat
    Sinus bradycardia
    Skin discolouration
    Skin discomfort
    Throat tightness
    Thrombosis (injection site)
    Upper respiratory tract congestion


    It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

    The following number will direct the caller to the relevant local centre (0844) 892 0111

    Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( ) or if this is unavailable at the backup site ( ).

    Further Information

    Last Full Review Date: July 2021

    Reference Sources

    Drugs in Pregnancy and Lactation: A Reference Guide to Fetal and Neonatal Risk, 10th edition (2015) ed. Briggs, G., Freeman, R. Wolters Kluwer Health, Philadelphia.

    Summary of Product Characteristics: Fabrazyme 5mg, powder for concentrate for solution for infusion. Sanofi Genzyme. Revised July 2021.
    Summary of Product Characteristics: Fabrazyme 35mg, powder for concentrate for solution for infusion. Sanofi Genzyme. Revised July 2021.

    NICE Evidence Services Available at: Last accessed: 20 July 2021.

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