Recombinant acid sphingomyelinase
Olipudase alfa
Brand names: Xenpozyme
Adult dose
Dose: Per BNF/SmPC; weight-based IV every 2 weeks
Route: IV
Frequency: q2w
Clinical pearls
- Acid sphingomyelinase deficiency (ASMD; Niemann-Pick A/B); specialist metabolic
Contraindications
- Severe hypersensitivity
Side effects
- Infusion reactions
- Pyrexia
- Hepatotoxicity
- Antibody development
Monitoring
- LFTs
- Antibody titres
Reference: BNF; SmPC; https://bnf.nice.org.uk/drugs/olipudase-alfa/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
Pathways
- Diabetic Ketoacidosis (DKA) · JBDS 2013 / Joint British Diabetes Societies; NICE NG17
- Adult Hypoglycaemia (Treated Diabetes) · JBDS-IP (2023): Hospital Management of Hypoglycaemia
- Adrenal Crisis · Society for Endocrinology Emergency Guidance (2024)
- Type 2 Diabetes Management · NICE NG28 2022
- Hyperthyroidism Management · BTA / ETA 2018
- Adrenal Insufficiency · Society of Endocrinology / ESE 2016