Enzyme replacement for Fabry disease
Agalsidase
A regular drip that replaces the missing enzyme in Fabry disease, an inherited lysosomal storage disorder.
What is Agalsidase?
Agalsidase is an enzyme replacement therapy for Fabry disease, an inherited condition in which a fatty substance builds up in the body because an enzyme is missing or not working, gradually affecting the kidneys, heart, nerves and skin. It is given as a slow drip into a vein, usually every couple of weeks and for life, at a specialist metabolic centre. It does not cure Fabry disease but helps control it and slow the damage. The most common problem is an infusion reaction, which the team manages with pre-medication and by slowing the drip.
Education and reference only. This is a plain-language guide to Agalsidase — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.
What it is
Agalsidase is a man-made version of alpha-galactosidase A, the enzyme that people with Fabry disease lack or cannot make properly. Fabry disease is a rare inherited 'lysosomal storage' disorder, meaning a fatty substance that should be broken down instead piles up inside cells, slowly harming the kidneys, heart, nerves and skin. Two forms are used in the UK, agalsidase alfa (Replagal) and agalsidase beta (Fabrazyme), both given as a drip into a vein. It is started and supervised by a specialist metabolic team.
How it works
Agalsidase supplies the working enzyme that people with Fabry disease are missing, so the fatty substance that has been building up in cells can be broken down. Given regularly into a vein, it helps clear these deposits from tissues such as the kidneys, heart and blood vessels, which can ease symptoms and slow further damage over time. Because the body keeps making the fatty substance, the enzyme has to be replaced again and again, which is why treatment is lifelong rather than a one-off.
Company & origin
Originated / developed by: Specialist (Takeda / Sanofi).
A laboratory-made copy of a missing enzyme, given by drip in UK specialist metabolic centres to treat the inherited condition Fabry disease.
Practical use
How to take Agalsidase
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- It is given as a slow drip into a vein at a specialist centre, usually every couple of weeks, by trained staff.
- You may be given pre-medication, such as antihistamine or paracetamol, to reduce the chance of an infusion reaction.
- Tell the staff straight away if you feel flushed, hot, shivery, sick or short of breath during the drip, as the rate can be slowed.
- Keep your regular appointments, as missing infusions allows the fatty substance to build up again.
- Some people who are stable can be trained to have the drip at home, but only once the specialist team agrees.
Weighing it up
Advantages & disadvantages of Agalsidase
Advantages
- Replaces the missing enzyme and can clear harmful deposits, helping to control Fabry disease and slow organ damage.
- Can ease symptoms such as pain and may help protect the kidneys and heart when started early.
- Allows many people to be treated at a specialist centre and, once stable, sometimes at home.
Disadvantages
- Does not cure the condition and must be given by drip for life.
- Commonly causes infusion reactions that need pre-medication and careful monitoring.
- Works best before permanent organ damage, so benefit is more limited once the disease is advanced.
Practical use
Good to know
Agalsidase controls Fabry disease but does not cure it, so the drips continue for life and are arranged through a specialist metabolic centre. The most common issue is an infusion reaction, with things like flushing, fever, chills, headache or feeling sick during or soon after the drip; the team often gives pre-medication and slows the rate to prevent or settle these. Over time some people make antibodies to the enzyme, which the team keeps an eye on. Treatment usually works best when started before there is a lot of permanent organ damage, so early diagnosis matters, and family members may be offered testing because Fabry disease is inherited. Some people are eventually trained to have their infusions at home once they are stable.
Who should not take it / use with caution
- People who have had a severe allergic reaction to agalsidase should not receive it again unless specialists advise.
- It is used with extra care in those who have developed antibodies that reduce its effect or worsen reactions.
- Any other illnesses, pregnancy or breastfeeding should be discussed with the metabolic team before continuing.
Monitoring
- Regular checks of kidney function, the heart and overall symptoms to see how the disease is responding.
- Watching for infusion reactions during and after each drip and for antibodies to the enzyme over time.
- Ongoing specialist review to decide whether home infusions are suitable and whether treatment is still helping.
Side effects
- Infusion reactions such as flushing, fever, chills, headache, nausea or feeling tired are the most common.
- Less commonly, tingling, dizziness, a fast heartbeat or a rash during or after the drip.
- Rarely, a serious allergic reaction, which is why infusions are given where staff can respond quickly.
Key interactions
- No major routine medicine interactions are well established, but always tell the team everything you take.
- Certain medicines that affect the heart or kidneys are reviewed in the wider care of Fabry disease.
- Other treatments are coordinated by the specialist centre alongside the enzyme infusions.
Available as: A drip (infusion) into a vein, given at a specialist centre or sometimes at home.
Answers
Agalsidase: frequently asked questions
Will agalsidase cure my Fabry disease?
No. It replaces the missing enzyme and helps control the disease and slow damage, but it does not cure it, so the drips are needed for life.
Why do I feel unwell during the drip?
Infusion reactions such as flushing, fever or feeling sick are common; the team often gives pre-medication and can slow the drip to settle them.
How often will I need the infusion?
It is usually given every couple of weeks at a specialist centre, though your team will confirm the schedule that is right for you.
Can I have it at home?
Some people who are stable are trained to have their infusions at home, but only once the specialist team agrees it is safe.
Should my family be tested?
Fabry disease is inherited, so relatives may be offered testing and advice; your specialist team can arrange this.
Authoritative sources
- BNF
- NICE CKS
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