alglucosidase alfa at a glance
Medicine class
Enzyme replacement therapy
Licensed use
Confirmed Pompe disease in adults and children of all ages
Administration
Intravenous infusion under specialist supervision
Key safety issue
Infusion-associated and allergic reactions can occur during or shortly after administration
What is alglucosidase alfa used for?
Licensed for long-term enzyme replacement therapy in people with confirmed Pompe disease.
Infantile-onset Pompe disease
Late-onset Pompe disease
Adults and children of all ages
How does alglucosidase alfa work?
Pompe disease causes deficient acid alpha-glucosidase activity, an enzyme that breaks down glycogen inside lysosomes. Alglucosidase alfa supplies a recombinant version of this enzyme, helping cells clear lysosomal glycogen and supporting cardiac, respiratory and skeletal muscle function.
alglucosidase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 50 mg
How to take alglucosidase alfa
It is prepared from a powder and given through a drip into a vein by trained healthcare professionals. The infusion is monitored and may be slowed or stopped if a reaction develops. Home infusion may be considered by the specialist team for selected people who have tolerated treatment well, with appropriate supervision and emergency arrangements.
Report new symptoms during the infusion or in the following hours immediately.
Do not change home-infusion arrangements without the treating team.
alglucosidase alfa side effects
Common or expected effects
- Infusion-associated reactions
- Hives, rash, itching or flushing
- Fever or chills
- Cough or faster breathing
- Nausea, vomiting or diarrhoea
- Headache, dizziness or tremor
- Fast heartbeat or raised blood pressure
- Tiredness, muscle pain or muscle spasms
- Infusion-site pain or reaction
Get urgent medical advice
- Severe allergic reaction or anaphylaxis, including breathing difficulty, throat tightness, swelling, low blood pressure or collapse
- Bronchospasm, low blood oxygen or respiratory distress
- Severe ulcerative or necrotising skin lesions
- Generalised or leg swelling with protein loss in urine, which may indicate nephrotic syndrome
- Serious abnormal heart rhythm or cardiac arrest
alglucosidase alfa in pregnancy
Experience during pregnancy is limited and animal studies identified reproductive toxicity. Use during pregnancy only when the person’s clinical condition requires it, after discussion with the Pompe specialist and obstetric team.
alglucosidase alfa while breastfeeding
Only very small amounts are expected to enter breast milk, and an effect on the infant is not expected because oral absorption is poor. Breastfeeding may be considered with specialist advice; temporary interruption after an infusion may also be discussed as a precaution.
alglucosidase alfa interactions
Formal interaction studies have not been performed. Interactions through cytochrome P450 metabolism are unlikely, but the specialist team should review all medicines.
Immunosuppressive medicines
May further increase the risk of serious respiratory infection in people with Pompe disease and require additional vigilance.
Common questions about alglucosidase alfa
Answers are fully visible for fast scanning and source review.
What is it used to treat?
It is licensed as long-term enzyme replacement therapy for confirmed Pompe disease in adults and children of all ages.
Can it be given at home?
Home infusion may be considered for selected people who tolerate treatment well and have appropriate professional supervision, training and emergency arrangements.
Can reactions happen after the infusion?
Yes. Reactions usually occur during the infusion or within the following hours, although delayed flu-like reactions lasting several days have also been reported.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.