agalsidase alfa at a glance
Medicine class
Lysosomal enzyme replacement therapy
Main use
Confirmed Fabry disease caused by alpha-galactosidase A deficiency
Administration
Intravenous infusion after dilution, supervised by a specialist service
Home treatment
May be considered after specialist assessment and training when infusions are well tolerated
What is agalsidase alfa used for?
Agalsidase alfa is licensed for enzyme replacement therapy in people with a confirmed diagnosis of Fabry disease, an inherited alpha-galactosidase A deficiency.
Fabry disease
How does agalsidase alfa work?
Fabry disease allows globotriaosylceramide, also called Gb3 or GL-3, to accumulate inside cells. Agalsidase alfa replaces the deficient lysosomal enzyme and breaks down this material, reducing its accumulation in several cell types. It does not correct the underlying genetic change.
agalsidase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 1 mg/mL
How to take agalsidase alfa
Agalsidase alfa is diluted and given into a vein using a line with an integral filter. It must not run through the same intravenous line as another medicine.
Treatment should be supervised by a clinician experienced in Fabry disease or inherited metabolic disorders.
Home infusion or trained self-administration may be considered only after specialist assessment.
A responsible adult should be present for self-administration.
Do not change the prescribed amount or infusion rate without the treating team's agreement.
agalsidase alfa side effects
Common or expected effects
- Infusion reactions, including chills, fever, flushing, headache, nausea, breathlessness, tremor, itching or tiredness
- Headache, dizziness, tingling, numbness or nerve pain
- Nausea, vomiting, abdominal pain or diarrhoea
- Rash
- Muscle, joint, back or limb pain
- Palpitations or chest discomfort
Get urgent medical advice
- Severe allergic reaction or anaphylaxis, including throat or tongue swelling and breathing difficulty
- Significant heart rhythm disturbance, severe chest pain, myocardial ischaemia or heart failure symptoms
- Severe infusion reaction with marked breathlessness, faintness or cardiovascular symptoms
agalsidase alfa in pregnancy
Pregnancy experience is very limited, so prescribing requires caution and discussion with the Fabry specialist and maternity team. A small published case series reported no adverse maternal or child outcomes, but it is too small to establish safety.
agalsidase alfa while breastfeeding
It is not known whether agalsidase alfa passes into human milk. Breastfeeding and treatment should therefore be discussed with the specialist team, considering the mother's clinical need and the benefits of breastfeeding.
agalsidase alfa interactions
Agalsidase alfa is unlikely to have conventional cytochrome P450 interactions, but some medicines may inhibit its intracellular enzyme activity.
Chloroquine
Should not be co-administered because it may inhibit intracellular alpha-galactosidase activity.
Amiodarone
Should not be co-administered because it may inhibit intracellular alpha-galactosidase activity.
Benoquin (monobenzone)
Should not be co-administered because it may inhibit intracellular alpha-galactosidase activity.
Gentamicin
Common questions about agalsidase alfa
Answers are fully visible for fast scanning and source review.
Is agalsidase alfa the same as Replagal?
Agalsidase alfa is the active ingredient; Replagal is the brand name of the selected UK product.
Does agalsidase alfa cure Fabry disease?
No. It replaces deficient enzyme activity and helps reduce stored Gb3, but it does not correct the inherited genetic change.
How is agalsidase alfa given?
It is 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.