Agalsidase alfa

How it works

It is a recombinant form of the human enzyme alpha-galactosidase A that replaces the deficient enzyme, allowing breakdown of accumulated globotriaosylceramide in tissues.

Prescribing in practice

• Infusion-related reactions are common, so it should be given under specialist supervision with measures to manage hypersensitivity available.
• It is administered by intravenous infusion on a regular basis and treatment is usually lifelong within a specialist Fabry service.
• Patients may develop antibodies to the enzyme; assess response and tolerability over time as set out in the SPC.

Monitoring

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

Counselling the patient

• Tell staff immediately if you feel flushed, feverish, itchy or breathless during your infusion.
• Attend your regular infusions and specialist follow-up to track how the condition is responding.
• Carry information about your diagnosis and treatment for use in an emergency.

Evidence & guidelines

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