cipaglucosidase alfa at a glance
Medicine class
Recombinant acid alpha-glucosidase enzyme replacement therapy.
Licensed use
Late-onset Pompe disease in adults, in combination with miglustat.
Administration
Given as an intravenous infusion under specialist supervision.
Home infusion
May be considered after infusions have been consistently well tolerated and the person is medically stable.
What is cipaglucosidase alfa used for?
It is licensed only as combination therapy.
Enzyme replacement treatment for adults with late-onset Pompe disease caused by acid alpha-glucosidase deficiency.
It must be used with the enzyme stabiliser miglustat.
How does cipaglucosidase alfa work?
Pompe disease causes glycogen to accumulate because acid alpha-glucosidase is deficient. Cipaglucosidase alfa replaces this enzyme and enters muscle-cell lysosomes, where it breaks down glycogen. Miglustat stabilises it while it circulates in the blood.
cipaglucosidase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 105 mg
How to take cipaglucosidase alfa
A trained healthcare professional prepares cipaglucosidase alfa and gives it into a vein. It must be administered with miglustat; follow the specialist team’s instructions. Home infusion is suitable only when approved and organised by the treating team.
Tell staff promptly about symptoms during or shortly after the infusion.
Dizziness, sleepiness or low blood pressure can occur; use caution when driving or using machinery after treatment.
Other medicines must not be infused through the same intravenous line.
cipaglucosidase alfa side effects
Common or expected effects
- Headache, dizziness, sleepiness or altered taste
- Flushing, blood-pressure changes or a fast heartbeat
- Breathlessness or cough
- Nausea, diarrhoea, abdominal discomfort, bloating or vomiting
- Rash, hives, itching or increased sweating
- Muscle or joint pain, muscle spasms or weakness
- Fatigue, fever, chills, chest discomfort, pain or swelling around the infusion site
Get urgent medical advice
- Anaphylaxis or another severe allergic reaction
- Severe worsening of breathing or heart function during an infusion, particularly with existing cardiorespiratory impairment
- Possible systemic immune-complex reactions, including severe skin changes or symptoms suggesting kidney involvement
cipaglucosidase alfa in pregnancy
There are no clinical pregnancy data for cipaglucosidase alfa with miglustat, and reproductive toxicity occurred in animal studies. The combination is not recommended during pregnancy. Discuss contraception and any planned or suspected pregnancy with the specialist team.
cipaglucosidase alfa while breastfeeding
It is unknown whether cipaglucosidase alfa or miglustat enters human milk, and a risk to the infant cannot be excluded. The specialist and patient should decide whether to stop breastfeeding or interrupt treatment after weighing the benefits of each.
cipaglucosidase alfa interactions
Formal interaction studies have not been performed. Conventional CYP- or P-glycoprotein-mediated interactions are unlikely.
Miglustat
Required treatment partner that stabilises cipaglucosidase alfa; its contraindications and administration requirements must also be checked.
Other intravenous medicines
Do not mix them with cipaglucosidase alfa or infuse them through the same intravenous line because compatibility has not been established.
Common questions about cipaglucosidase alfa
Answers are fully visible for fast scanning and source review.
What is cipaglucosidase alfa used for?
It is licensed with miglustat as enzyme replacement therapy for adults with late-onset Pompe disease.
Why is it given with miglustat?
Miglustat stabilises cipaglucosidase alfa in the bloodstream, helping preserve enzyme activity before uptake into target tissues.
Can it be given at home?
Sometimes. Specialist-approved home infusion may be considered for medically stable people whose supervised infusions have been consistently well tolerated.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.