Section 16 · Monograph
Avalglucosidase alfa
Reviewed Nov 2026FDA label
NexviazymeEnzyme replacement (GAA)
- Forms
- IV: 100mg vial
- Dose
- Pompe disease: 20mg/kg IV every 2 weeks (40mg/kg if under 30kg)
- MOA
- Replaces acid alpha-glucosidase with enhanced M6P targeting
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
- Late-onset Pompe disease (lysosomal acid alpha-glucosidase deficiency) in patients 1 year of age and olderFDA-labeled
Mechanism of Action
- Recombinant human GAA enhanced with mannose-6-phosphate residues for improved muscle uptake and lysosomal glycogen clearance
Full Dosing
Adult / usual
- 20mg/kg IV every 2 weeks (40mg/kg every 2 weeks if under 30kg per product policies); infuse over about 4 hours with stepwise rate increases
- Premedication may reduce infusion reactions
- Do not combine with other GAA products
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
- Anaphylaxis and hypersensitivity
- Acute cardiorespiratory failure risk in susceptible patients
- Immunogenicity
Monitoring Parameters
- Close monitoring during infusion
- Cardiorespiratory status and fluid load in at-risk patients
- Clinical response (FVC, 6-minute walk)
Pregnancy & Lactation
- Limited data; use if benefit justifies risk
- Unknown whether present in human milk