agalsidase beta at a glance
Medicine class
Lysosomal enzyme replacement therapy
Licensed use
Confirmed Fabry disease in adults and people aged 8 years and over
Administration
Intravenous infusion supervised by a clinician experienced in inherited metabolic disease
Home treatment
May be considered after good infusion tolerance and specialist assessment
What is agalsidase beta used for?
UK-licensed use:
Long-term enzyme replacement therapy for confirmed Fabry disease caused by alpha-galactosidase A deficiency, in adults, children and adolescents aged 8 years and over.
How does agalsidase beta work?
Fabry disease causes reduced or absent alpha-galactosidase A activity, allowing GL-3 and related substances to accumulate in lysosomes. After infusion, agalsidase beta is taken into cells and their lysosomes, where it replaces enzyme activity and helps clear this stored material.
agalsidase beta preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 5 mg, 35 mg
How to take agalsidase beta
Agalsidase beta is not swallowed and food does not affect its administration. The powder is reconstituted, diluted with sodium chloride solution and given into a vein. Home infusion is only appropriate after specialist assessment, suitable training and established tolerance; treatment settings must not be changed without the clinical team.
Tell the infusion team promptly about symptoms occurring during the infusion or later that day.
Do not drive or operate machinery if dizziness, sleepiness, vertigo or fainting occurs during or shortly after treatment.
Home infusions should be recorded as instructed by the treating service.
agalsidase beta side effects
Common or expected effects
- Chills, fever or feeling cold
- Nausea or vomiting
- Headache
- Burning, tingling or other altered skin sensation
- Infusion reactions such as flushing, itching, rash, tiredness, muscle pain or abdominal discomfort
Get urgent medical advice
- Severe allergic reaction with swelling of the face or throat, breathing difficulty, wheezing, low oxygen, fainting or collapse
- Marked throat or chest tightness, bronchospasm, or a severe change in blood pressure during an infusion
agalsidase beta in pregnancy
Human pregnancy data are limited, so the UK product information advises avoiding agalsidase beta as a precaution. Published experience consists mainly of small reports with generally favourable outcomes and cannot establish safety; decisions about continuing treatment require individual specialist assessment.
agalsidase beta while breastfeeding
Agalsidase beta has been detected in human milk, and its effects on a breastfed infant are unknown. The specialist team should weigh the benefits of breastfeeding against the benefits of continuing treatment before either is stopped.
agalsidase beta interactions
Formal interaction studies have not been performed, but cytochrome P450 interactions are considered unlikely. The following combinations should be avoided because they may inhibit intracellular alpha-galactosidase A activity:
Chloroquine
There is a theoretical risk of reducing agalsidase beta activity.
Amiodarone
There is a theoretical risk of reducing agalsidase beta activity.
Benoquin (monobenzone)
There is a theoretical risk of reducing agalsidase beta activity.
Common questions about agalsidase beta
Answers are fully visible for fast scanning and source review.
What is agalsidase beta used for?
It is licensed as long-term enzyme replacement therapy for people with a confirmed diagnosis of Fabry disease who fall within the licensed age group.
Does agalsidase beta cure Fabry disease?
No. It replaces deficient enzyme activity and helps clear accumulated material, but it does not correct the inherited genetic change.
How is agalsidase beta given?
It is prepared from a powder, diluted and administered as an intravenous infusion under specialist supervision.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.