Alglucosidase Alfa

How it works

It replaces deficient lysosomal acid alpha-glucosidase, enabling breakdown of accumulated lysosomal glycogen and thereby limiting glycogen-related damage to cardiac and skeletal muscle.

Prescribing in practice

• Serious infusion-associated and hypersensitivity reactions, including anaphylaxis, can occur, so administer under supervision with resuscitation facilities available and consider premedication in at-risk patients.
• Administered by intravenous infusion at a specialist centre, typically on a regular recurring schedule.
• Infantile-onset patients, particularly those who are CRIM-negative, may develop anti-drug antibodies that can reduce efficacy.

Monitoring

Monitor for infusion-associated reactions during and after dosing, watch for cardiorespiratory compromise in infantile-onset disease, and consider periodic anti-drug antibody testing.

Counselling the patient

• Report any rash, breathing difficulty, fever, or feeling unwell during or after the infusion immediately.
• Treatment is long term and given by infusion in a specialist setting.

Evidence & guidelines

Enzyme replacement is the established disease-specific treatment for Pompe disease; consult the SPC and current prescribing references for administration and monitoring detail.