Skip to content
ClinCalc Pro
Menu
Calculators Drugs Pathways AI Dx Donate Get the App
Recombinant acid sphingomyelinase

Olipudase alfa

Brand names: Xenpozyme

Olipudase alfa is an enzyme replacement therapy used to treat the non-central-nervous-system manifestations of acid sphingomyelinase deficiency (Niemann-Pick disease types A/B and B) in children and adults.

Dosing — being independently re-sourced

ClinCalc Pro is rebuilding its dose data from primary open sources — the manufacturer SmPC (eMC), the WHO Model Formulary and other official references — under clinician review. This drug's structured dose is not yet published here. Confirm all doses against the product SmPC and your local formulary before prescribing.

Clinical monograph

How it works

It is a recombinant human acid sphingomyelinase that replaces the deficient enzyme, breaking down accumulated sphingomyelin in tissues.

Prescribing in practice

  • Infusion-associated and hypersensitivity reactions, including anaphylaxis, can occur, so it is given with gradual dose escalation and appropriate premedication and monitoring under specialist care.
  • Rapid breakdown of accumulated substrate can cause transient elevations in liver enzymes and inflammatory markers, requiring monitoring after dose increases.
  • It does not treat the neurological manifestations of the disease, so the central nervous system features are managed separately.

Monitoring

Monitor liver function tests, inflammatory markers and for infusion-related and hypersensitivity reactions, particularly around dose escalation.

Counselling the patient

  • This treatment is given as an intravenous infusion in a monitored setting.
  • Report any reaction such as rash, breathlessness or feeling unwell during or after the infusion.
  • Attend scheduled blood tests so the dose can be adjusted safely.

Evidence & guidelines

Olipudase alfa is an approved enzyme replacement therapy that improves pulmonary and haematological outcomes in acid sphingomyelinase deficiency.

Reference: SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

Calculators
Pathways