Formulary
Olipudase alfa: Indications, Dosing, Side Effects and Interactions
Olipudase alfa is an enzyme produced by recombinant DNA technology that is administered as enzyme replacement therapy for the treatment of acid...
MedNext Academy | 3 min read
Olipudase alfa: Indications, Dosing, Side Effects and Interactions
Olipudase alfa is an enzyme produced by recombinant DNA technology that is administered as enzyme replacement therapy for the treatment of acid...
Drug action
Olipudase alfa is an enzyme produced by recombinant DNA technology that is administered as enzyme replacement therapy for the treatment of acid sphingomyelinase deficiency (Niemann-Pick disease) type A/B or B, a lysosomal storage disorder affecting the normal function of tissues and organs such as the liver, spleen, lungs, heart, and brain.
Indications and dose
**Non-neurological manifestations of acid sphingomyelinase deficiency (Niemann-Pick disease) type A/B or B (under expert supervision)**
- **Adult** (By Intravenous Infusion): Initially 100 micrograms/kg for 1 dose, subsequent doses to be administered every 2 weeks according to dose escalation regimen until maintenance phase is reached after 14 weeks-consult product literature; usual maintenance 3 mg/kg every 2 weeks.
**Non-neurological manifestations of acid sphingomyelinase deficiency (Niemann-Pick disease) type A/B or B (under expert supervision) for olipudase alfa**
- **Neonate** (By intravenous infusion): Initially 30 micrograms/kg for 1 dose, subsequent doses to be administered every 2 weeks according to dose escalation regimen until maintenance phase is reached after 16 weeks-consult product literature; usual maintenance 3 mg/kg every 2 weeks.
- **Child** (By intravenous infusion): Initially 30 micrograms/kg for 1 dose, subsequent doses to be administered every 2 weeks according to dose escalation regimen until maintenance phase is reached after 16 weeks-consult product literature; usual maintenance 3 mg/kg every 2 weeks.
Cautions
Infusion-related reactions Cautions, further information Infusion-related reactions Serious infusion-related reactions can occur and olipudase alfa should only be administered when appropriately trained staff and resuscitation facilities are available; these reactions may be minimised by pre-medication with an antihistamine, antipyretic, and corticosteroid. Patients should be closely monitored for signs of infusion-related reactions during and after administration, and managed appropriately-consult product literature. M
Side effects
Common or very common Angioedema; arthralgia; asthenia; chills; diarrhoea; dyspnoea; eye discomfort; eye erythema; fever; gastrointestinal discomfort; headache; hepatic pain; hypersensitivity; hypotension; myalgia; nausea; pain; palpitations; respiratory disorders; skin reactions; tachycardia; throat complaints; vasodilation; vomiting
Pregnancy
Avoid unless potential benefit outweighs risk-toxicity in animal studies. M
Breast feeding
Specialist sources indicate use with caution-no information available. Large molecular weight suggests limited excretion into milk and drug molecule likely to be partially destroyed in the infant's gastro-intestinal tract.
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Clinical governance
**Author:** MedNext Clinical Team. **Clinical reviewer:** Dr Shameer Deen, MBBS, MS, MRCS. **Sources:** BNF, Indian Pharmacopoeia, CIMS India. **Correction:** Report errors at support@mednext.academy.
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