Kanuma (sebelipase alfa)

The recommended initial dosage of sebelipase alfa in patients with rapidly progressive lysosomal acid lipase deficiency presenting within the first 6 months of life is 1 mg/kg as an IV infusion once weekly. For infants who do not achieve an optimal clinical response, the dosage may be increased to 3 mg/kg once weekly. For infants who continue to experience a suboptimal response at the 3 mg/kg once weekly dosage, the dosage may be increased to 5 mg/kg once weekly. Suboptimal response is defined as the presence of any of the following: poor growth, deteriorating biochemical markers, or persistent or worsening organomegaly.

The recommended dosage of sebelipase alfa in adults and pediatric patients with lysosomal acid lipase deficiency is 1 mg/kg as an IV infusion once every other week. For patients who do not achieve an optimal clinical response, the dosage may be increased to 3 mg/kg once every other week. Suboptimal response is defined as the presence of any of the following: poor growth, deteriorating biochemical markers, and/or lipid metabolism parameters.

Sebelipase alfa is administered by IV infusion only. The drug should be infused over at least 2 hours. A longer infusion time should be considered in patients receiving sebelipase alfa dosages greater than 1 mg/kg or in patients who have experienced hypersensitivity reactions. A shorter infusion time of 1 hour may be considered, if tolerated, in patients receiving a 1-mg/kg dose of the drug.

Sebelipase alfa should be administered with a low-protein-binding infusion set equipped with an inline, low-protein-binding 0.2-mcm filter.

Commercially available sebelipase alfa injection should be stored at 2-8degreesC in the original carton to protect from light; the vials should not be frozen or shaken.

No dosing information is available for this tab.

Formal drug interaction studies have not been performed to date with sebelipase alfa.

There are no contraindication interactions for this drug.

There are no severe interactions for this drug.

There are no moderate interactions for this drug.

*None.

There are no contraindication contraindications for this drug.

There are no severe contraindications for this drug.

• No disease contraindications

The most common adverse reactions (>=30%) in infants with rapidly progressive lysosomal acid lipase deficiency presenting within the first 6 months of life receiving sebelipase alfa include diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, and urticaria.

The most common adverse reactions (>=8%) in pediatric and adult patients with lysosomal acid lipase deficiency receiving sebelipase alfa include headache, fever, oropharyngeal pain, nasopharyngitis, asthenia, constipation, and nausea.

More Frequent

• Anemia

Less Frequent

• Acute respiratory distress syndrome
• Anaphylaxis
• Dyspnea
• Hypersensitivity drug reaction
• Hypertension
• Laryngeal edema
• Tachypnea

More Frequent

• Cough
• Diarrhea
• Fever
• Headache disorder
• Hyperlipidemia
• Hypertriglyceridemia
• Pharyngitis
• Rhinitis
• Sore throat
• Urticaria
• Vomiting

Less Frequent

• Acute abdominal pain
• Agitation
• Chest discomfort
• Chills
• Constipation
• Eczema
• Edema
• Erythema
• Eyelid edema
• General weakness
• Irritability
• Pallor
• Pruritus of skin
• Rhinorrhea
• Tachycardia

Rare or Very Rare

• Ocular redness

No enhanced messaging available.

Safety and efficacy of sebelipase alfa have been established in pediatric patients 1 month of age and older in clinical studies. Clinical trials of sebelipase alfa included 56 pediatric patients (1 month to <18 years of age).

Clinical data on use of sebelipase alfa in pregnant women are insufficient to identify a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes; animal studies have not suggested any evidence of adverse embryofetal effects.

It is not known whether sebelipase alfa is distributed into human milk. Effects of sebelipase alfa on the breastfed infant or on milk production are also unknown. The known benefits of breast-feeding should be considered along with the mother's clinical need for sebelipase alfa and any potential adverse effects of the drug or disease on the infant.

Clinical studies of sebelipase alfa did not include patients 65 years of age and older. It is not known whether geriatric patients respond differently than younger patients.