← Search & browse all drugs
ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Enzymes
sebelipase alfa
A16AB14
Forms & Strengths
- Intravenous solution: 2 mg/mL in a single-use vial
Adult Dosing
- Lysosomal acid lipase (LAL) deficiency: 1 mg/kg intravenously once every other week
- Weight-based dosing; round to the nearest mg
- May increase up to 3 mg/kg every other week in patients with suboptimal clinical response
Pediatric Dosing
- Rapidly progressive LAL deficiency presenting in infants: Initial dose 1 mg/kg IV once weekly, may be increased up to 3 mg/kg weekly based on clinical response
- Pediatric patients (children and adolescents): 1 mg/kg IV once every other week, may be increased up to 3 mg/kg every other week
Indications
- Treatment of patients with lysosomal acid lipase (LAL) deficiency (Wolman disease / Cholesteryl ester storage disease)
Mechanism of Action
- Recombinant human lysosomal acid lipase (rhLAL) binds to cell-surface mannose-6-phosphate receptors, is internalized into lysosomes, and catalyzes the hydrolysis of cholesteryl esters and triglycerides to cholesterol and free fatty acids
Contraindications
- History of a severe hypersensitivity reaction, including anaphylaxis, to sebelipase alfa or excipients
Adverse Reactions
- Hypersensitivity reactions including anaphylaxis
- Headache
- Fever
- Nasopharyngitis
- Oropharyngeal pain
- Constipation
- Rhinorrhea
- Hypotension
- Tachycardia
Drug Interactions
- No formal clinical drug-drug interaction studies have been conducted
- Potential for hypersensitivity reactions may be exacerbated by concurrent immunosuppressive therapy
Curated Content: Needs Vetting Before Put to Clinical Use