Enzyme Replacement Therapy (Next-generation — Pompe Disease)

Avalglucosidase Alfa

Brand names: Nexviazyme

Adult dose

Dose: 20 mg/kg every 2 weeks IV

Route: Intravenous infusion

Frequency: Every 2 weeks

Clinical pearls

Next-generation ERT for Pompe disease (glycogen storage disease type II) — engineered with high mannose-6-phosphate content for improved lysosomal targeting
COMET trial: superior to alglucosidase alfa in treatment-naive infantile-onset Pompe
NICE TA813 (2023): avalglucosidase alfa recommended for infantile-onset Pompe disease and for late-onset Pompe when eligible and where alglucosidase alfa not adequate
Enhanced uptake into muscle cells compared with first-generation alglucosidase alfa
Specialist use only — NHS England Highly Specialised Technology (HST)
Eligibility criteria include evidence of disease progression or non-response on alglucosidase alfa

Contraindications

Life-threatening hypersensitivity to avalglucosidase alfa not manageable

Side effects

Infusion-related reactions (very common)
Anaphylaxis
Hypersensitivity
Pyrexia
Fatigue
Nausea

Interactions

No significant drug interactions known

Monitoring

Pulmonary function tests (FVC) 6-monthly
Motor function assessments (6MWT, Walton scale)
Anti-avalglucosidase antibody titres
Infusion parameters
Renal function (Pompe-related)

Reference: BNF; NICE TA813 (Avalglucosidase alfa for treating Pompe disease, 2023); COMET trial (Lancet 2022); NHS England HST pathway; https://bnf.nice.org.uk/drugs/avalglucosidase-alfa/. Verify against your local formulary and the latest BNF before prescribing.

Curated clinical cross-links plus same-class fallbacks.

Calculators

Pathways