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Recombinant α-galactosidase A (ERT)

Agalsidase beta

Brand names: Fabrazyme

Agalsidase beta is an enzyme replacement therapy used for the long-term treatment of Fabry disease, an inherited lysosomal storage disorder.

Dosing — being independently re-sourced

ClinCalc Pro is rebuilding its dose data from primary open sources — the manufacturer SmPC (eMC), the WHO Model Formulary and other official references — under clinician review. This drug's structured dose is not yet published here. Confirm all doses against the product SmPC and your local formulary before prescribing.

Clinical monograph

How it works

It is a recombinant form of human alpha-galactosidase A that replaces the deficient enzyme and catalyses the breakdown of globotriaosylceramide that accumulates in Fabry disease.

Prescribing in practice

  • Infusion-associated reactions are common, so administer under specialist supervision with facilities to manage hypersensitivity, premedicating where appropriate.
  • It is given by regular intravenous infusion as lifelong treatment within a specialist Fabry service.
  • Antibody formation can occur and may affect response; evaluate tolerability and disease control over time as set out in the SPC.

Monitoring

Monitor for infusion reactions during administration and review cardiac, renal and overall disease progression periodically within the specialist service.

Counselling the patient

  • Report flushing, fever, chills, itching or breathlessness during your infusion straight away.
  • Keep to your scheduled infusions and specialist reviews so treatment can be monitored.
  • Carry details of your diagnosis and treatment for emergencies.

Evidence & guidelines

Use of enzyme replacement therapy for Fabry disease is guided by specialist commissioning and lysosomal storage disorder service protocols.

Reference: NHS England LSDU; SmPC Fabrazyme; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.