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Inborn errors of metabolism
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Generic Name and Formulations:

Sebelipase alfa 20mg/10mL; soln for IV infusion after dilution; preservative-free.

Select therapeutic use:

Indications for KANUMA:

Treatment of lysosomal acid lipase (LAL) deficiency.

Adults and Children:

<1 month: not established. Give as IV infusion over ≥2hrs; consider further prolonging infusion time for the 3mg/kg dose or if a hypersensitivity reaction occurs. May consider a 1hr infusion for the 1mg/kg dose in those who tolerate the infusion. ≥1 month: 1mg/kg once every other week. If rapidly progressive within first 6 months of life: initially 1mg/kg once weekly; increase to 3mg/kg once weekly if optimal response not achieved. May pre-treat with antipyretics and/or antihistamines.


Monitor for hypersensitivity reactions during and after infusion; interrupt or lower infusion rate based on severity. Discontinue immediately and treat if severe hypersensitivity including anaphylaxis occurs. Eggs or egg products allergy. Pregnancy. Nursing mothers.

Pharmacological Class:

Hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme.

Adverse Reactions:

Diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, urticaria, headache, oropharyngeal pain, asthenia, constipation, nausea.

Generic Availability:


How Supplied:

Single-use vials—1

KANUMA 10ml of 2mg/ml vials (Qty:1)

appx. price $10107.00