About sebelipase alfa
Lysosomal acid lipase replacement enzyme
Used for
- Long-term enzyme replacement for lysosomal acid lipase deficiency, including Wolman disease and cholesteryl ester storage disease.
Usual adult dose
1 mg/kg by intravenous infusion every other week; some patients may need 3 mg/kg every other week.
Usual dose in children
Infants with rapidly progressive disease: 1 mg/kg weekly, increased to 3 mg/kg weekly if needed. Other children: 1 mg/kg every other week; some need 3 mg/kg.
Common side effects
- Headache
- Fever
- Diarrhea
- Infusion-related reactions, including rash or breathing difficulties
Important warnings
- Serious allergic reactions, including anaphylaxis, can occur during or after infusion.
- Infusion reactions may require slowing or stopping treatment.
- Antibodies to the medicine may develop and affect treatment response.
- Infusions require monitoring by healthcare professionals.
Pregnancy
Human pregnancy data are limited; use only when a clinician judges treatment benefits outweigh potential risks.
Breastfeeding
It is unknown whether the medicine passes into breast milk; discuss potential benefits and risks with a clinician.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.