• Lysosomal acid lipase deficiency.

Contraindicated in:

• Hypersensitivity to eggs.

Use Cautiously in:

• OB: Safety not established in pregnancy
• Lactation: Safety not established in breastfeeding
• Pedi: Children <1 mo (safety and effectiveness not established)
• Geri: Older adults (safety and effectiveness not established).

CV: tachycardia.

EENT: nasopharyngitis, rhinitis, sneezing.

GI: diarrhea, oropharyngeal pain, vomiting, constipation, nausea.

Hemat: anemia.

Metab: ↑LDL cholesterol, ↑triglycerides.

MS: hypotonia.

Neuro: headache, anxiety.

Resp: cough, hypoxemia.

Misc: fever, HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS, URTICARIA).

Drug-Drug:

• None reported.

• Solution for injection: 2 mg/mL;

Rapidly Progressive Lysosomal Acid Lipase Deficiency Presenting within First 6 Mo of Age

• IV (Infants): 1 mg/kg once weekly; may ↑ to 3 mg/kg once weekly if suboptimal response (poor growth, deteriorating biochemical markers, or persistent or worsening organomegaly) achieved; may ↑ to 5 mg/kg once weekly if suboptimal response continues to be achieved.

Lysosomal Acid Lipase Deficiency in Children and Adults

• IV (Adults and Children): 1 mg/kg every other wk; may ↑ to 3 mg/kg every other wk if suboptimal response (poor growth, deteriorating biochemical markers [e.g., ALT, AST], and/or parameters of lipid metabolism [e.g., LDL cholesterol, triglycerides].

Kanuma

• A recombinant form of lysosomal acid lipase, it binds to cell surface receptors via glycans expressed on the protein and is subsequently internalized into lysosomes. It then catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol, and free fatty acids.

• Improved survival in infants.
• Improved lipids in adults and children.

Therapeutic Classification: n/a

Pharmacologic Classification: enzymes

Absorption: IV administration results in complete bioavailability.

Distribution: Well distributed to tissues.

Metabolism/Excretion: Unknown.

Half-life: 5.4–6.6 min.

(plasma concentrations)

• Explain purpose and side effects of sebelipase alfa to patient and/or caregiver.
• Advise patient and/or caregiver to readPatient Information before starting therapy.
• Advise patient and/or caregiver to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
• Teach patient and/or caregiver the signs and symptoms of an hypersensitivity reaction; if any occur, seek immediate medical care.
• Advise patient and/or caregiver to report known allergies to eggs or egg products prior to treatment.
• Advise patient and/or caregiver if a dose is missed to notify a health care professional.
• Rep: Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding.

se-be-LYE-pase AL-fa

NDC Code^*

• 25682- Alexion Pharmaceuticals, Inc.
  • 25682-007- KANUMA
    • 25682-007-01- 1 VIAL, GLASS in 1 CARTON (25682-007-01) > 10 mL in 1 VIAL, GLASS