Avalglucosidase Alfa

How it works

It provides an exogenous form of the deficient lysosomal enzyme acid alpha-glucosidase, which is taken up by cells and degrades accumulated lysosomal glycogen in muscle.

Prescribing in practice

• Serious infusion-associated and hypersensitivity reactions, including anaphylaxis, can occur, so infusions require appropriate monitoring and resuscitation facilities.
• It is administered by intravenous infusion at regular intervals as long-term therapy.
• Patients may develop antibodies to the enzyme, which can affect response and tolerability.

Monitoring

Observe patients closely during and after infusions for hypersensitivity and infusion-associated reactions.

Counselling the patient

• This is a long-term treatment requiring regular infusions in a supervised setting.
• Report any rash, breathlessness, flushing or feeling unwell during or after an infusion.

Evidence & guidelines

Enzyme replacement therapy is the established disease-specific treatment for Pompe disease and is supported by clinical trial evidence in this rare condition.