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Sebelipase Alfa (Monograph)

Brand name: Kanuma
Drug class: Enzymes
Chemical name: Cholesterol (human gene LIPA) esterase,
Molecular formula: C1968H2945N507O551S15
CAS number: 1276027-63-4


Biosynthetic (recombinant DNA origin) form of human lysosomal acid lipase.

Uses for Sebelipase Alfa

Lysosomal Acid Lipase Deficiency

Treatment of lysosomal acid lipase deficiency (designated an orphan drug by FDA for this use).

Survival benefit demonstrated in patients with rapidly progressive infantile-onset disease (i.e., Wolman disease).

In pediatric patients and adults with late-onset disease (i.e., cholesterol ester storage disease), improvements in disease-related lipid and hepatic abnormalities demonstrated.

Effects on cardiovascular morbidity and mortality and progression of liver disease not established.

Sebelipase Alfa Dosage and Administration


IV Administration

For solution compatibility information, see Compatibility under Stability.

Administer by IV infusion.

Administer using an inline, low-protein-binding 0.2-µm filter.

Contains no preservatives; use immediately following preparation. (See Storage under Stability.) Discard any unused portions.


Prior to administration, must dilute commercially available sebelipase alfa injection concentrate.

Determine number of vials to be diluted based on patient’s weight and recommended dose of 1 or 3 mg/kg.

Remove required number of vials from refrigerator and allow to reach room temperature; do not shake. Withdraw appropriate dose from vials and dilute with 0.9% sodium chloride injection to prepare a final concentration of 0.1–1.5 mg/mL.

See Table 1 for recommended total infusion volumes based on patient's weight and prescribed dose.

For patients presenting within the first 6 months of life who do not achieve optimal clinical response with a dose of 1 mg/kg.

Table 1: Total Infusion Volumes

Patient Weight (kg)

Total Infusion Volume (mL) for 1-mg/kg Dose

Total Infusion Volume (mL) for 3-mg/kg Dose
















Mix diluted solution gently; do not shake. The solution should appear clear to slightly opalescent and colorless to slightly colored; thin translucent fibers or particles may be present. Do not administer if solution appears cloudy or if other particulate matter observed.

Rate of Administration

Infuse over at least 2 hours. Consider longer infusion times in patients receiving a dose of 3 mg/kg or in patients who experience hypersensitivity reactions. Consider shorter infusion time of 1 hour, if tolerated, in patients receiving a dose of 1 mg/kg.


Pediatric Patients

Lysosomal Acid Lipase Deficiency
Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency Presenting Within the First 6 Months of Life

1 mg/kg once weekly. In patients not achieving optimal clinical response, may increase to 3 mg/kg once weekly.

Pediatric Patients with Lysosomal Acid Lipase Deficiency

1 mg/kg once every 2 weeks.


Lysosomal Acid Lipase Deficiency

1 mg/kg once every 2 weeks.

Special Populations

No special population dosage recommendations at this time.

Cautions for Sebelipase Alfa



Sensitivity Reactions


Hypersensitivity reactions, including anaphylaxis, reported. Generally during or within 4 hours of the infusion. Anaphylaxis reported as early as the sixth infusion to as late as 1 year after treatment initiation.

Ensure that appropriate medical support is readily available during administration. Closely observe patients during and after administration.

Manage hypersensitivity reactions based on severity. If anaphylaxis or other severe hypersensitivity reaction occurs, discontinue sebelipase alfa immediately and initiate appropriate medical treatment. For hypersensitivity reactions of lesser severity, may temporarily interrupt infusion, lower infusion rate, and/or administer antihistamines, antipyretics, and/or corticosteroids. If infusion is interrupted, resume at a slower rate; subsequently increase rate as tolerated.

To prevent subsequent reactions in patients experiencing hypersensitivity, may consider premedication with antipyretics and/or antihistamines. Consider risks and benefits of retreatment following a severe hypersensitivity reaction. If drug is readministered, monitor patient with appropriate resuscitation measures available.

Egg Allergy

Consider risks and benefits of treatment in patients with known systemic hypersensitivity reactions to eggs or egg products.

Antibody Formation

Development of antibodies, including neutralizing antibodies, to sebelipase alfa reported.

Hypersensitivity reactions and possible reduced efficacy (decreased growth) observed in antibody-positive infants. No clear association between development of antibodies and efficacy in adults and pediatric patients.

Specific Populations


No available data in pregnant women; animal studies have not suggested any evidence of adverse embryofetal effects.


Not known whether sebelipase alfa is distributed into milk. Consider known benefits of breast-feeding along with mother's need for sebelipase alfa and any potential adverse effects of the drug or disease on the infant.

Pediatric Use

Safety and efficacy established in pediatric patients ≥1 month of age.

Geriatric Use

Not studied in patients ≥65 years of age. Not known whether geriatric patients respond differently than younger patients.

Common Adverse Effects

Infants with rapidly progressive lysosomal acid lipase deficiency presenting within the first 6 months of life: Diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, urticaria.

Pediatric and adult patients with lysosomal acid lipase deficiency: Headache, fever, oropharyngeal pain, nasopharyngitis, abdominal pain, asthenia, constipation, nausea.

Drug Interactions

Formal drug interaction studies not performed to date.

Sebelipase Alfa Pharmacokinetics

Pharmacokinetic profile of sebelipase alfa is based on data from adults and pediatric patients; pharmacokinetics not characterized in infants.



Nonlinear pharmacokinetics; greater than dose-proportional increase in exposure observed at doses of 1–3 mg/kg.

No substantial accumulation following repeated dosing.

Peak plasma concentrations occur approximately 1.1–1.3 hours after a dose in pediatric patients and adults.


Increased LDL-cholesterol and triglyceride concentrations initially observed within the first 2–4 weeks; such parameters subsequently decreased to below pretreatment levels within 8 weeks of treatment. Decreased ALT concentrations typically observed within 2 weeks of treatment.

Special Populations

Pharmacokinetic profile similar between adolescents and adults.



Not known whether sebelipase alfa distributes into milk.



Approximately 6 minutes.

Half-life similar across pediatric and adult patients of all age groups receiving 1 mg/kg once every other week.




Injection for IV Infusion

2–8°C. Protect from light. Do not freeze; do not shake.

Following dilution, 2–8°C for ≤24 hours if protected from light. Do not freeze.



Solution Compatibility1


Sodium chloride 0.9%


Advice to Patients


Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Sebelipase Alfa


Dosage Forms


Brand Names



Injection concentrate, for IV infusion

2 mg/mL (20 mg)



AHFS DI Essentials™. © Copyright 2024, Selected Revisions March 22, 2017. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.

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