sebelipase alfa (Rx)

Brand and Other Names:Kanuma
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Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

solution for injection

  • 20mg/10mL per vial (2mg/mL)

Lysosomal Acid Lipase Deficiency

Indicated for enzyme replacement in patients with lysosomal acid lipase (LAL) deficiency

1 mg/kg IV infusion administered every other week (see Administration)

Dosage Forms & Strengths

solution for injection

  • 20mg/10mL per vial (2mg/mL)

Lysosomal Acid Lipase Deficiency

Indicated for enzyme replacement in patients with lysosomal acid lipase (LAL) deficiency

1 mg/kg IV infusion administered every other week (see Administration)

Rapidly progressive LAL deficiency presenting within the first 6 months of life

  • Starting dose: 1 mg/kg IV infusion administered once weekly
  • May increase to 3 mg/kg once weekly for patients who do not achieve an optimal clinical response with the starting dose
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Adverse Effects

>10%

Age <6 months

  • Diarrhea (67%)
  • Vomiting (67%)
  • Fever (56%)
  • Rhinitis (56%)
  • Anemia (44%)
  • Cough (33%)
  • Nasopharyngitis (33%)
  • Urticaria (33%)

Pediatric and adults

  • Headache (28%)
  • Fever (25%)
  • Oropharyngeal pain (17%)
  • Nasopharyngitis (11%)

1-10%

Pediatric and adults

  • Asthenia (8%)
  • Constipation (8%)
  • Nausea (8%)
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Warnings

Contraindications

None

Cautions

Hypersensitivity reactions, including anaphylaxis, have been reported as early as the sixth infusion and as late as 1 yr after treatment initiation; symptoms during the infusion may include chest discomfort, conjunctival injection, dyspnea, generalized and itchy rash, hyperemia, swelling of eyelids, rhinorrhea, severe respiratory distress, tachycardia, tachypnea, and urticaria; monitor closely during IV infusion; may need to slow the infusion rate or temporarily interrupt the infusion; discontinue if severe signs and symptoms occur; treat with appropriate medical support

Product 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 risks and benefits in patients with known systemic hypersensitivity reactions to eggs or egg products

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Pregnancy

Pregnancy

There are no available data 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

Lactation

Unknown if distributed in human breast milk

Pregnancy Categories

A:Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B:May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C:Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D:Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X:Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA:Information not available.

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Pharmacology

Mechanism of Action

Recombinant form of the human lysosomal acid lipase (LAL) enzyme

LAL deficiency is an autosomal recessive lysosomal storage disorder characterized by a genetic defect resulting in a marked decrease or loss in activity of 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 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-c

Absorption

Peak plasma time: 1.1-1.3 hr

Peak plasma concentration: 490-957 ng/mL

AUC: 942-1861 ng·hr/mL

Distribution

Vd: 3.6-5.4 L

Elimination

Half-life: 5.4-6.6 minutes

Clearance: 31.1-38.2 L/hr

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Administration

IV Preparation

Determine number of vials needed for dose based on patient’s weight; round to the next whole vial and remove the required number of vials from the refrigerator to allow them to reach room temperature

Determine the volume of the calculated dose and the necessary volume of 9% NaCl for dilution; the infusion volume should be based on the prescribed dose and should be prepared to a final concentration of 0.1-1.5 mg/mL (see total volume sections listed below)

Mix gently by inversion; do not shake the vials or the prepared infusion

Inspected visually for particulate matter and discoloration prior to administration; the solution should be a clear to slightly opalescent, colorless to slightly colored solution; thin, translucent particles or fibers may be present in the vials or diluted solution

Do not use if the solution is cloudy or if other particulate matter is observed

Vials are for single use only; discard any unused product

Contains no preservatives; therefore, product should be used immediately after dilution; if this is not possible, may store refrigerated (see Storage)

1 mg/kg dose total infusion volume

  • Drug volume plus 0.9% NaCl for dilution
  • 1-10.9 kg: 10 mL
  • 11-24.9 kg: 25 mL
  • 25-49.9 kg: 50 mL
  • 50-99.9 kg: 100 mL
  • 100-120.9 kg: 250 mL

3 mg/kg dose total infusion volume

  • Drug volume plus 0.9% NaCl for dilution
  • 1-10.9 kg: 25 mL
  • 11-24.9 kg: 50 mL
  • 25-49.9 kg: 100 mL
  • 50-99.9 kg: 250 mL
  • 100-120.9 kg: 500 mL

IV Administration

Administer IV infusion using a low-protein binding infusion set with an in-line, low-protein binding 0.2 micron filter

Infuse over at least 2 hr

Consider further prolonging the infusion time for patients receiving the 3 mg/kg dose or those who have experienced hypersensitivity reactions (see Cautions)

A 1-hr infusion may be considered for those patients receiving the 1 mg/kg dose who tolerate the infusion

Storage

Unopened vials

  • Refrigerate between 2-8°C (36-46°F) in original carton to protect from light
  • Do not shake or freeze the vials

Diluted solution

  • If immediate use is not possible, the diluted product may be stored up to 24 hr in the refrigerator at 2-8°C (36-46°F)
  • Do not freeze or shake
  • Protect from light
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Formulary

FormularyPatient Discounts

Adding plans allows you to compare formulary status to other drugs in the same class.

To view formulary information first create a list of plans. Your list will be saved and can be edited at any time.

Adding plans allows you to:

  • View the formulary and any restrictions for each plan.
  • Manage and view all your plans together – even plans in different states.
  • Compare formulary status to other drugs in the same class.
  • Access your plan list on any device – mobile or desktop.

The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
NC NOT COVERED – Drugs that are not covered by the plan.
Code Definition
PA Prior Authorization
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
QL Quantity Limits
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
ST Step Therapy
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
OR Other Restrictions
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.