Section 16 · Monograph
Alglucosidase alfa
Reviewed Nov 2026FDA label
LumizymeEnzyme replacement (GAA)
- Forms
- IV: 50mg vial
- Dose
- Pompe disease: 20mg/kg IV every 2 weeks
- MOA
- Replaces acid alpha-glucosidase to degrade lysosomal glycogen
Black Box Warning
- Risk of anaphylaxis, hypersensitivity and immune-mediated reactions, and risk of acute cardiorespiratory failure from fluid overload in patients with compromised cardiac or respiratory function — appropriate medical support must be available during infusion
Indications
- Pompe disease (acid alpha-glucosidase deficiency) in patients 1 month of age and olderFDA-labeled
Mechanism of Action
- Recombinant human acid alpha-glucosidase that restores lysosomal glycogen degradation in muscle
Full Dosing
Adult / usual
- 20mg/kg IV every 2 weeks, infused over about 4 hours with stepwise rate increases
- Premedication with antihistamines, antipyretics and/or corticosteroids may reduce infusion reactions
- Not interchangeable with other GAA products; do not combine with Nexviazyme or Pombiliti
Contraindications
- No absolute contraindications listed; severe hypersensitivity requires stopping the infusion and weighing rechallenge
Drug Interactions
- No formal interaction studies
Adverse Effects
- Infusion-associated reactions (fever, rash, flushing, tachycardia, cough)
- Anaphylaxis and hypersensitivity
- Acute cardiorespiratory failure risk in susceptible patients
- Immunogenicity (anti-drug antibodies, especially CRIM-negative patients)
Monitoring Parameters
- Close monitoring during infusion
- Cardiorespiratory status and fluid load in at-risk patients
- Anti-drug antibodies and clinical response (6-minute walk, FVC)
Pregnancy & Lactation
- Limited data; use if benefit justifies risk
- Unknown whether present in human milk