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Sebelipase Alfa

Medically reviewed by Last updated on Jun 17, 2020.


(se be LYE pase AL fa)

Index Terms

  • SBC-102

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Kanuma: 20 mg/10 mL (10 mL) [contains albumin human, egg white (egg protein)]

Brand Names: U.S.

  • Kanuma

Pharmacologic Category

  • Enzyme, Replacement Therapy


Sebelipase alfa binds to cell surface receptors via glycans expressed on the protein and is subsequently internalized into lysosomes. Sebelipase alfa catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol, and free fatty acids. In patients with lysosomal acid lipase (LAL) deficiency, replacement with sebelipase alfa, a recombinant form of LAL, results in improvement in disease-related hepatic and lipid parameters.


Pediatric patients: Vd:

4 to 11 years: 3.6 ± 3 L

12 to 17 years: 5.4 ± 2.4 L

Adults: Vd: 5.3 ± 1.6 L

Onset of Action

LDL-c and triglycerides reduction: Within 8 weeks; a transient increase in these values occurs during first 2 to 4 weeks of treatment.

ALT reduction: Within 2 weeks.

Time to Peak

Serum: Children ≥4 years, Adolescents, and Adults: Mean range: 1.1 to 1.3 hours

Duration of Action

Effects on ALT partially reverse 3 weeks after treatment discontinuation (Balwani 2013).

Half-Life Elimination

Children ≥4 years, Adolescents, and Adults: Mean range: 5.4 to 6.6 minutes

Use: Labeled Indications

Lysosomal acid lipase deficiency: Treatment of patients with lysosomal acid lipase (LAL) deficiency


There are no contraindications listed within the manufacturer's labeling.

Canadian labeling: Life-threatening hypersensitivity to sebelipase or any component of the formulation

Dosing: Adult

Lysosomal acid lipase (LAL) deficiency: IV: 1 mg/kg every other week

Dosing: Geriatric

LAL deficiency: Refer to adult dosing; has not been studied.

Dosing: Pediatric

Lysosomal acid lipase (LAL) deficiency; rapidly progressive presenting within the first 6 months of life: Infants and Children: IV: Initial: 1 mg/kg/dose once weekly; may increase to 3 mg/kg/dose once weekly if response not optimal; in the trials, the higher 3 mg/kg dose was initiated between 4 and 88 weeks of therapy (median: 11 weeks)

LAL deficiency: Infants, Children, and Adolescents: IV: 1 mg/kg/dose every other week

Dosing adjustment for toxicity: All patients:

Mild or moderate hypersensitivity reaction: Interrupt infusion; consider resuming at slower infusion rate.

Severe hypersensitivity reaction or anaphylaxis: Immediately discontinue infusion.

Dosing: Adjustment for Toxicity

Mild or moderate hypersensitivity reaction: Interrupt infusion; consider resuming at slower infusion rate.

Severe hypersensitivity reaction or anaphylaxis: Immediately discontinue infusion.


Determine number of vials necessary for dose. After allowing vial(s) to reach room temperature, further dilute calculated dose volume with NS up to the manufacturer-recommended total infusion volume based on patient weight (refer to manufacturer’s product labeling) to a final concentration range of 0.1 to 1.5 mg/mL. Mix gently by inversion. Do not shake vials or prepared infusion. Reconstituted solution should be clear to slightly opalescent, colorless to slightly colored solution. Thin, translucent particles or fibers may be present in vials or diluted solution. Vials are for single use only.


IV: Administer diluted solution as an IV infusion using low-protein-binding infusion set with in-line, low-protein-binding 0.2 micron filter. Infuse over at least 2 hours; may consider 1-hour infusion for those patients receiving 1 mg/kg dose who tolerate a 2-hour infusion. Consider further prolonging infusion time for 3 mg/kg dose or if hypersensitivity reaction occurs.


Store intact vials under refrigeration between 2°C to 8°C (36°F to 46°F) in original carton to protect from light. Do not shake or freeze. If not used immediately, reconstituted product may be stored up to 24 hours in the refrigerator.

Drug Interactions

There are no known significant interactions.

Adverse Reactions

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.

Frequency not always defined. Incidences are reported for children, adolescents, and adult patients unless specified otherwise.

Cardiovascular: Chest discomfort, oxygen saturation decreased (infants), tachycardia (infants)

Central nervous system: Headache (28%), anxiety, hypotonia (infants)

Dermatologic: Urticaria (infants: 33%)

Endocrine & metabolic: Increase in LDL cholesterol (81%; mean increase of 18% at 2 and 4 weeks postinitiation), increased serum triglycerides (58%; mild increases that are transient)

Gastrointestinal: Diarrhea (infants: 67%), vomiting (infants: 67%), constipation (8%), nausea (8%), retching (infants)

Hematologic & oncologic: Anemia (infants: 44%)

Hypersensitivity: Hypersensitivity reaction (20%; infants: 64%; children, adolescents, and adults: 13%), anaphylaxis (3%; may be delayed as late as 1 year after treatment initiation)

Immunologic: Immunogenicity (infants: ≤57%; neutralizing and may affect drug efficacy: 29%; children, adolescents, and adults: 14%; neutralizing without any association on drug efficacy: 6%)

Neuromuscular & skeletal: Weakness (8%)

Respiratory: Rhinitis (infants: 56%), cough (infants: 33%), nasopharyngitis (infants: 33%; children and adult patients: 11%), oropharyngeal pain (17%), sneezing (infants)

Miscellaneous: Fever (infants: 56%; children and adult patients: 25%)


Concerns related to adverse effects:

• Anaphylaxis/hypersensitivity reactions: Hypersensitivity reactions have occurred during infusion and within 4 hours after the infusion. Anaphylaxis has occurred as early as the sixth infusion and as late as 1 year after treatment initiation. During infusion, appropriate medical treatment should be immediately available. Depending on severity of hypersensitivity reaction, management may include temporary interruption, lowering infusion rate, and/or administration of antihistamines, antipyretics, and/or corticosteroids. If interrupted, the infusion may be resumed at a slower rate with increases as tolerated. Pretreatment with antipyretics and/or antihistamines may prevent subsequent reactions in those cases where symptomatic treatment was required. If anaphylaxis or severe hypersensitivity occurs, immediately discontinue infusion and initiate appropriate medical treatment.

• Antibody formation: Patients have developed anti-drug antibodies (ADA) to sebelipase alfa and may be more likely to experience hypersensitivity reactions. Some patients with neutralizing antibodies experienced decreased growth velocity. No clear association exists between the development of ADA and decreased efficacy in patients.

Dosage form specific issues:

• Allergy to egg or egg products: Sebelipase alfa is produced in egg whites of genetically engineered chickens; consider the risks and benefits in patients with known systemic hypersensitivity reactions to eggs or egg products.

Monitoring Parameters

Signs and symptoms of hypersensitivity reaction (during infusion and for at least 4 hours after completion of infusion); lipid panel; anti-drug antibody assessment; hepatic function (ALT).

Pregnancy Considerations

Adverse events were not observed in animal reproduction studies. Sebelipase alfa is a recombinant form of lysosomal acid lipase (LAL), an essential enzyme required for lipid metabolism (Burton 2015; Shirley 2015).

Patient Education

What is this drug used for?

• It is used to treat a rare health problem called lysosomal acid lipase (LAL) deficiency.

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Cough

• Nasal irritation

• Throat irritation

• Constipation

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Chest pain

• Fast heartbeat

• Shortness of breath

• Eyelid swelling

• Runny nose

• Fast breathing

• Abdominal pain

• Agitation

• Chills

• Severe diarrhea

• Swelling

• Severe headache

• Severe dizziness

• Passing out

• Irritability

• Severe nausea

• Severe vomiting

• Pale skin

• Severe loss of strength and energy

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine’s uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.