Enzyme replacement therapy

Agalsidase beta (أجالسيداز بيتا)

Agalsidase beta is a man-made copy of the enzyme alpha-galactosidase A, which is missing in Fabry disease, a rare inherited condition in which a fatty substance builds up in the kidneys, heart, nerves and blood vessels. It is given as a drip into a vein long term under a specialist.

What it is used for

  • Long-term treatment of confirmed Fabry disease to replace the missing enzyme
  • Slowing the build-up of the fatty substance and damage to the kidneys and heart, and easing nerve pain in some patients

How it is used

  • It is given as a drip into a vein by a nurse or doctor, and may be given at home after training if your specialist decides
  • It is given at regular intervals set by your specialist, and treatment is usually lifelong
  • You may be given medicines before the drip to prevent infusion reactions
  • Attend regular kidney, heart and nerve check-ups

Possible side effects

  • Infusion reactions such as chills, fever, flushing, headache and nausea
  • Tiredness or pain in the arms and legs
  • Dizziness or palpitations
  • Rarely, a severe allergic reaction

Who should not use it

  • Severe allergy to the medicine or any of its ingredients

Before and while using it

  • Tell the nurse at once if you feel breathless or get a rash or facial swelling during or after the drip
  • The body can make antibodies to the medicine that increase infusion reactions or reduce its effect
  • People with severe heart disease need close monitoring during the drip
  • Do not switch to another enzyme product unless your specialist decides

Medicine interactions

  • Chloroquine, amiodarone and gentamicin may reduce the enzyme's activity and are not recommended with it; tell your doctor about all your medicines

Pregnancy and breastfeeding

  • Information is limited; your specialist decides about continuing it in pregnancy or breastfeeding

How to store it

  • Vials are kept in the fridge and prepared in hospital or as your care team instructs

Get medical help straight away if

  • Your face, lips or tongue swell, or you find it hard to breathe.
  • You get a severe rash, blistering or peeling skin.
  • A side effect worries you or doesn’t go away: contact your doctor or pharmacist.

Questions about this medicine?

Talk to a doctor by video, or send your question by text.

Source: Standard international medicine references

This guide is general information and doesn’t replace advice from your doctor or pharmacist. Don’t start, stop or change a medicine without asking them, and always read the leaflet in the pack.