Formulary
Sebelipase alfa: Indications, Dosing, Side Effects and Interactions
Sebelipase alfa is a recombinant human lysosomal acid lipase used as enzyme replacement therapy in lysosomal acid lipase deficiency (such as Wolman disease).
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Sebelipase alfa: Indications, Dosing, Side Effects and Interactions
Sebelipase alfa is a recombinant human lysosomal acid lipase used as enzyme replacement therapy in lysosomal acid lipase deficiency (such as Wolman disease).
Drug action
Sebelipase alfa is a recombinant human lysosomal acid lipase used as enzyme replacement therapy in lysosomal acid lipase deficiency (such as Wolman disease).
Indications and dose
**Lysosomal acid lipase deficiency (under expert supervision)**
- **Adult** (By Intravenous Infusion): 1 mg/kg every 2 weeks, then increased if necessary to 3 mg/kg every 2 weeks.
**Lysosomal acid lipase deficiency [rapidly progressive; Wolman disease] (under expert supervision) for sebelipase alfa**
- **Neonate** (By intravenous infusion): Initially 1 mg/kg every week for at least 4 doses, then increased if necessary to 3 mg/kg every week, then increased if necessary to 5 mg/kg every week, further dose adjustments can be made according to response; doses higher than 7.5 mg/kg once weekly have not been studied, alternatively initially 3 mg/kg every week, then increased if necessary to 5 mg/kg every week, further dose adjustments can be made according to response; doses higher than 7.5 mg/kg once weekly have not been studied.
- **Child 1-5 months** (By intravenous infusion): Initially 1 mg/kg every week for at least 4 doses, then increased if necessary to 3 mg/kg every week, then increased if necessary to 5 mg/kg every week, further dose adjustments can be made according to response; doses higher than 7.5 mg/kg once weekly have not been studied, alternatively initially 3 mg/kg every week, then increased if necessary to 5 mg/kg every week, further dose adjustments can be made according to response; doses higher than 7.5 mg/kg once weekly have not been studied.
**Lysosomal acid lipase deficiency (under expert supervision) for sebelipase alfa**
- **Child 6 months-17 years** (By intravenous infusion): 1 mg/kg every 2 weeks, then increased if necessary to 3 mg/kg every 2 weeks.
Cautions
Hypersensitivity reactions Cautions, further information Hypersensitivity reactions Hypersensitivity reactions, including anaphylaxis, have been reported, usually during or within 4 hours of an infusion; anaphylaxis has occurred during infusion as long as 1 year after treatment initiation. Kanuma should only be administered by appropriately trained staff, and patients should be observed for 1 hour after the initial infusion or after any dose increase; manage hypersensitivity reactions by temporarily interrupting the infusion, reducing the rate of infusion and/or treatment with antihistamines, antipyretics, and/or corticosteroids. If a severe reaction occurs, stop infusion immediately and start appropriate medical treatment; caution is advised on re-administration. Pre-treatment with antipyretics and/or antihistamines may prevent subsequent reactions. M
Side effects
Common or very common Chest discomfort; diarrhoea; dizziness; dyspnoea; fatigue; fever; gastrointestinal discomfort; hyperaemia; hypersensitivity; hypotension; skin reactions; tachycardia
Pregnancy
Avoid as a precaution (no information available). M
Breast feeding
Avoid (no information available). M
Medicinal forms
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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