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Generic Name: Sebelipase Alfa
Class: Enzymes


Sebelipase alfa is an enzyme.

Uses for Kanuma

Sebelipase alfa has the following uses:

Sebelipase alfa is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of lysosomal acid lipase (LAL) deficiency.1

Kanuma Dosage and Administration


Sebelipase alfa is available in the following dosage form(s) and strength(s):

Injection: 20 mg/10 mL (2 mg/mL) solution in single-use vials.1


It is essential that the manufacturer’s labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

Patients with rapidly progressive LAL deficiency presenting within the first 6 months of life: The recommended starting dosage is 1 mg/kg as an intravenous infusion once weekly. For patients who do not achieve an optimal clinical response, increase to 3 mg/kg once weekly.1

Pediatric and adult patients with LAL deficiency: The recommended dosage is 1 mg/kg as an intravenous infusion once every other week.1

Administration Instructions:1

  • Infuse over at least 2 hours.1

  • Consider further prolonging the infusion time for the 3 mg/kg dose or if a hypersensitivity reaction occurs.1

  • Consider a 1-hour infusion for the 1 mg/kg dose in patients who tolerate the infusion.1

Cautions for Kanuma




Hypersensitivity Reactions Including Anaphylaxis

Hypersensitivity reactions, including anaphylaxis, have been reported in sebelipase alfa-treated patients. In clinical trials, 3 of 106 (3%) patients treated with sebelipase alfa experienced signs and symptoms consistent with anaphylaxis. These patients experienced reactions during infusion with signs and symptoms including chest discomfort, conjunctival injection, dyspnea, generalized and itchy rash, hyperemia, swelling of eyelids, rhinorrhea, severe respiratory distress, tachycardia, tachypnea, and urticaria. Anaphylaxis has occurred as early as the sixth infusion and as late as 1 year after treatment initiation.1

In clinical trials, 21 of 106 (20%) sebelipase alfa-treated patients, including 9 of 14 (64%) infants and 12 of 92 (13%) pediatric patients, 4 years and older, and adults, experienced signs and symptoms either consistent with or that may be related to a hypersensitivity reaction. Signs and symptoms of hypersensitivity reactions, occurring in two or more patients, included abdominal pain, agitation, fever, chills, diarrhea, eczema, edema, hypertension, irritability, laryngeal edema, nausea, pallor, pruritus, rash, and vomiting. The majority of reactions occurred during or within 4 hours of the completion of the infusion. Patients were not routinely pre-medicated prior to infusion of sebelipase alfa in these clinical trials.1

Due to the potential for anaphylaxis, appropriate medical support should be readily available when sebelipase alfa is administered. If anaphylaxis occurs, immediately discontinue the infusion and initiate appropriate medical treatment. Observe patients closely during and after the infusion. Inform patients of the signs and symptoms of anaphylaxis, and instruct them to seek immediate medical care should signs and symptoms occur.1

The management of hypersensitivity reactions should be based on the severity of the reaction and may include temporarily interrupting the infusion, lowering the infusion rate, and/or treatment with antihistamines, antipyretics, and/or corticosteroids. If interrupted, the infusion may be resumed at a slower rate with increases as tolerated. Pre-treatment with antipyretics and/or antihistamines may prevent subsequent reactions in those cases where symptomatic treatment was required. If a severe hypersensitivity reaction occurs, immediately discontinue the infusion and initiate appropriate medical treatment.1

Consider the risks and benefits of re-administering sebelipase alfa following a severe reaction. Monitor patients, with appropriate resuscitation measures available, if the decision is made to re-administer the product.1

Hypersensitivity to Eggs or Egg Products

Sebelipase alfa is produced in the egg whites of genetically engineered chickens. Patients with a known history of egg allergies were excluded from the clinical trials. Consider the risks and benefits of treatment with sebelipase alfa in patients with known systemic hypersensitivity reactions to eggs or egg products.1

Specific Populations


Risk Summary: There are no available data on sebelipase alfa in pregnant women to inform any drug-associated risk. Animal reproductive studies conducted with sebelipase alfa showed no evidence of embryolethality, fetotoxicity, teratogenicity, or abnormal early embryonic development at dosages up to 164 and 526 times the human dosage of 1 mg/kg every other week (based on AUC) in rats and rabbits, respectively.1 The background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.1

Animal Data: Sebelipase alfa administered during the period of organogenesis to rats (on gestation days 6, 9, 12, 15, and 17) and rabbits (on gestation days 7, 10, 13, 16, and 19) at intravenous doses up to 60 and 50 mg/kg, respectively, (approximately 164 and 526 times the human AUC of 1387 ng•h/mL at 1 mg/kg dose administered once every other week, respectively) did not cause any adverse effects on embryofetal development. A pre- and postnatal development study in rats showed no evidence of adverse effects on pre- and postnatal development at intravenous doses (administered on gestation days 6, 9, 12, 15, 18, and 20 and days 4, 7, 10, 14, and 17 postpartum) of sebelipase alfa up to 60 mg/kg/day (approximately 164 times the human AUC of 1387 ng•h/mL at 1 mg/kg dose administered once every other week).1


There are no data on the presence of sebelipase alfa in human milk, the effects on the breastfed infant, or the effects on milk production. It is not known if sebelipase alfa is present in animal milk. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for sebelipase alfa and any potential adverse effects on the breastfed infant from sebelipase alfa or from the underlying maternal condition.1

Pediatric Use

Safety and effectiveness of sebelipase alfa have been established in pediatric patients aged 1 month and older. Clinical trials with sebelipase alfa were conducted in 56 pediatric patients (range 1 month to <18 years old).1

Geriatric Use

Clinical trials of sebelipase alfa did not include any patients aged 65 years old and older. It is not known whether they respond differently than younger patients.1

Common Adverse Effects

The most common adverse reactions are:1

  • Patients with rapidly progressive disease presenting within the first 6 months of life (≥30%): diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, and urticaria.1

  • Pediatric and adult patients (≥8%): headache, fever, oropharyngeal pain, nasopharyngitis, asthenia, constipation, and nausea.1

Drug Interactions

Specific Drugs

It is essential that the manufacturer’s labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:

Please see product labeling for drug interaction information.1


Mechanism of Action

LAL deficiency is an autosomal recessive lysosomal storage disorder characterized by a genetic defect resulting in a marked decrease or loss in activity of the lysosomal acid lipase (LAL) enzyme. The primary site of action of the LAL enzyme is the lysosome, where the enzyme normally causes the breakdown of lipid particles including LDL-c. Deficient LAL enzyme activity results in progressive complications due to the lysosomal accumulation of cholesteryl esters and triglycerides in multiple organs, including the liver, spleen, intestine, and the walls of blood vessels. The resulting lipid accumulation in the liver may lead to increased liver fat content and progression of liver disease, including fibrosis and cirrhosis. Lipid accumulation in the intestinal wall leads to malabsorption and growth failure. In parallel, dyslipidemia due to impaired degradation of lysosomal lipid is common with elevated LDL-c and triglycerides and low HDL-cholesterol (HDL-c).1

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.1

Advice to Patients

Hypersensitivity Reactions, including Anaphylaxis

Advise patients and caregivers that reactions related to administration and infusion may occur during and after sebelipase alfa treatment, including life-threatening anaphylaxis and severe hypersensitivity reactions. Inform patients of the signs and symptoms of anaphylaxis and hypersensitivity reactions, and have them seek immediate medical care should signs and symptoms occur.1

Additional Information

AHFS First Release. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.


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, Solution, Concentrate

2 mg /1 mL


Alexion Pharmaceuticals Inc.

AHFS Drug Information. © Copyright 2017, Selected Revisions September 15, 2016. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.


1. Alexion Pharmaceuticals, Inc. KANUMA (Sebelipase alfa) INTRAVENOUS prescribing information. 2015 Dec.