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Enzyme Replacement Therapy (Lysosomal Storage Disorder)

Alglucosidase Alfa

Brand names: Myozyme

Adult dose

Dose: 20 mg/kg every 2 weeks
Route: Intravenous infusion
Frequency: Every 2 weeks (consult product SmPC for infusion rate)

Clinical pearls

  • Enzyme replacement therapy for Pompe disease (glycogen storage disease type II — acid maltase deficiency)
  • Both classic infantile-onset and late-onset forms may benefit
  • NICE TA321 recommends for late-onset Pompe disease (non-infantile)
  • Pre-medicate with antihistamines, antipyretics, corticosteroids to reduce infusion reactions
  • High-antibody titre patients may have reduced clinical response
  • Specialist use only — prescribed via NHS England Highly Specialised Technologies programme

Contraindications

  • Life-threatening hypersensitivity to alglucosidase alfa or excipients (if not manageable)

Side effects

  • Infusion-related reactions (flushing, urticaria, pyrexia, tachycardia) — common
  • Anaphylaxis (rare but serious)
  • Anti-drug antibody formation — may reduce efficacy
  • Fatigue
  • Nausea
  • Muscle pain

Interactions

  • No significant pharmacokinetic drug interactions known
  • Immunosuppressants — sometimes used to reduce antibody formation (specialist decision)

Monitoring

  • Pulmonary function tests (FVC, MIP, MEP) 6-monthly
  • Motor function (6-minute walk test, Walton scale) at baseline and every 6 months
  • Anti-alglucosidase antibody titres at 3 months, then periodically
  • Infusion parameters and vital signs during each infusion

Reference: BNF; NICE TA321 (Alglucosidase alfa for late-onset Pompe disease, 2014); NHS England HST pathway for Pompe disease; https://bnf.nice.org.uk/drugs/alglucosidase-alfa/. Verify against your local formulary and the latest BNF before prescribing.

Related

Curated clinical cross-links plus same-class fallbacks.

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