avalglucosidase alfa at a glance
Medicine class
Recombinant enzyme replacement therapy
Licensed use
Pompe disease caused by acid alpha-glucosidase deficiency
How it is given
Intravenous infusion prepared and administered by trained healthcare professionals
Home treatment
May be considered after infusions have been well tolerated and suitable support and training are established
What is avalglucosidase alfa used for?
Avalglucosidase alfa is licensed as enzyme replacement therapy for Pompe disease, also called acid alpha-glucosidase deficiency.
Treatment of infantile-onset and late-onset Pompe disease under specialist care.
How does avalglucosidase alfa work?
Pompe disease causes glycogen to accumulate inside lysosomes because acid alpha-glucosidase activity is deficient. Avalglucosidase alfa supplies a recombinant version of this enzyme. Its mannose-6-phosphate groups help cells take it into lysosomes, where it breaks down glycogen.
avalglucosidase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 100 mg
How to take avalglucosidase alfa
Avalglucosidase alfa is not taken by mouth. A healthcare professional reconstitutes and dilutes the powder, then gives it through a drip into a vein. The infusion is monitored and adjusted according to tolerance.
An antihistamine, antipyretic or corticosteroid may be given beforehand to reduce infusion reactions.
Tell staff immediately about symptoms during the infusion or in the hours afterwards.
Home infusion requires specialist assessment, tolerated supervised infusions, training and appropriate clinical support.
avalglucosidase alfa side effects
Common or expected effects
- Headache
- Nausea or vomiting
- Itching, rash or hives
- Fatigue, chills, fever or flu-like symptoms
- Dizziness or sleepiness
- Diarrhoea or abdominal discomfort
- Flushing or blood-pressure changes
- Infusion-site pain
Get urgent medical advice
- Difficulty breathing, wheezing or low blood oxygen
- Swelling of the lips, tongue, throat, face or eyelids
- Severe hives, chest pressure, faintness or collapse
- Blue or grey lips or skin
- Severe infusion reaction or anaphylaxis
- Sudden worsening of heart or breathing problems, particularly in people vulnerable to fluid overload
avalglucosidase alfa in pregnancy
Human pregnancy data are unavailable, so safety cannot be established. Use requires specialist assessment that the expected maternal benefit outweighs potential risks to the fetus.
avalglucosidase alfa while breastfeeding
It is unknown whether avalglucosidase alfa enters human milk or affects the breastfed child. The potential benefits and risks should be assessed before treatment while breastfeeding.
avalglucosidase alfa interactions
Formal interaction studies have not been performed. Cytochrome P450-mediated interactions are considered unlikely because avalglucosidase alfa is a recombinant human protein.
Other intravenous medicines
Avalglucosidase alfa must not be mixed with other medicines or infused through the same intravenous line.
Common questions about avalglucosidase alfa
Answers are fully visible for fast scanning and source review.
What is avalglucosidase alfa used to treat?
It is licensed as enzyme replacement therapy for people with Pompe disease.
How does it help in Pompe disease?
It supplies acid alpha-glucosidase activity inside lysosomes, helping cells break down accumulated glycogen.
Can it be given at home?
Sometimes. Home infusion may be considered after supervised infusions have been well tolerated and trained staff, procedures and emergency support are available.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.