Sebelipase Alfa
Sebelipase Alfa is a recombinant form of the lysosomal acid lipase (LAL) enzyme used as enzyme replacement therapy for patients with Lysosomal Acid Lipase (LAL) deficiency. This rare genetic disorder prevents the body from properly breaking down cholesteryl esters and triglycerides, leading to their accumulation in various tissues and organs.
A recombinant enzyme therapy that replaces deficient lysosomal acid lipase in patients with LAL deficiency, a rare genetic storage disorder.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- A16AB14
- RxNorm
- 1726975
- Generic
- Sebelipase Alfa
- Class
- atc-a16
- Brands
- Kanuma
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 1-3 mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1 mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1-3 mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
11 groupspregnancy
first trimesteruse only if benefits outweigh risks
continue treatment if pregnancy occurs only if clearly needed, discuss risks with healthcare provider
regular prenatal monitoring and fetal ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
second trimesteruse only if benefits outweigh risks
continue treatment if ongoing disease control is necessary
regular prenatal monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesteruse only if benefits outweigh risks
discuss with physician prior to delivery planning
regular prenatal monitoring
Use with caution and monitor for adverse effects.
lactation
breastfeedingno specific adjustment
decision to breastfeed should be made in consultation with healthcare provider, consider infant benefits
monitor infant for any unusual symptoms
pediatric_age
infants1 mg/kg once weekly via IV infusion
approved for infants with LAL-D, including rapidly progressive form, may start from birth
regular monitoring of growth, liver function, and lipid parameters
pediatric_age
children adolescents1 mg/kg once weekly via IV infusion
approved for children and adolescents with LAL-D
regular monitoring of growth, liver function, lipid profile, and developmental milestones
geriatric
elderlyno specific adjustment required
standard dosing applies, monitor renal and hepatic function
standard monitoring as per individual patient status
renal_impairment
mild moderateno specific dose adjustment required
no dose adjustment needed for mild to moderate renal impairment
routine clinical monitoring
renal_impairment
severeuse with caution
limited data available, clinical judgment required
increased monitoring of renal function
Use with caution and monitor for adverse effects.
hepatic_impairment
mild moderateno specific dose adjustment required
drug is indicated for treatment of LAL-D which affects liver, monitor liver function
regular liver function tests
hepatic_impairment
severeuse with caution
limited data available, benefits should be weighed against potential risks
close monitoring of liver function and clinical status
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval date for Kanuma
Drug class
Therapeutic classification
Mechanism of action
Biological activity
Primary indication
Therapeutic indication
Treatment of LAL deficiency (also known as Wolman disease in its infantile form and cholesteryl ester storage disease in its later-onset form), a rare autosomal recessive lysosomal storage disorder that causes harmful accumulation of fats in cells throughout the body.
As a recombinant form of the human LAL enzyme, sebelipase alfa binds to receptors on cell surfaces and is internalized into lysosomes, where it hydrolyzes cholesteryl esters and triglycerides that have accumulated due to the deficient or absent native enzyme activity.
Administered via intravenous infusion, typically weekly. Hypersensitivity reactions may occur during infusions. Contains no preservatives and must be used immediately after preparation. Safety and effectiveness established primarily through clinical studies in pediatric and adult patients with LAL deficiency.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy