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Generic name: sebelipase alfa
Drug class: Lysosomal enzymes

Medically reviewed by  A Ras MD.

What is Kanuma used for?

Kanuma is a prescription medicine that is used to treat a rare health problem called lysosomal acid lipase (LAL) deficiency.


Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL) that is a lysosomal glycoprotein enzyme produced by recombinant DNA technology in the egg white of eggs laid by genetically engineered chickens. Purified sebelipase alfa is a monomeric glycoprotein containing 6 N-linked glycosylation sites and has a molecular mass of approximately 55 kDa. The amino acid sequence for sebelipase alfa is the same as the amino acid sequence for human LAL. The specific activity of sebelipase alfa is 195 to 345 units/mg. One unit is the amount of enzyme activity that catalyzes the hydrolysis of 1 micromole of the synthetic substrate 4-methylumbelliferyl oleate per minute at 37°C under specified assay conditions.

KANUMA (sebelipase alfa) injection is supplied as a sterile, preservative-free, non-pyrogenic clear to slightly opalescent, colorless to slightly colored aqueous solution in single-dose vials for intravenous infusion. Each vial contains sebelipase alfa 20 mg/10 mL. Each mL of solution contains sebelipase alfa (2 mg), citric acid monohydrate (1.57 mg), Human Serum Albumin (10 mg), and trisodium citrate dihydrate (13.7 mg) at pH 5.9.

Mechanism of Action

LAL deficiency is an autosomal recessive lysosomal storage disorder characterized by a genetic defect resulting in a marked decrease or loss in activity of the lysosomal acid lipase (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 and triglycerides. 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 the 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-cholesterol (HDL-c).

Sebelipase alfa binds to cell surface receptors via glycans expressed on the protein and is subsequently internalized into lysosomes. Sebelipase alfa catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol and free fatty acids.

Before taking Kanuma, tell your doctor:

  • If you are allergic to Kanuma; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take Kanuma with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take Kanuma?

  • Tell all of your health care providers that you take Kanuma. This includes your doctors, nurses, pharmacists, and dentists.
  • Allergic reactions can happen with Kanuma. Some allergic reactions may happen some time after Kanuma has been given. Call your doctor right away if you have any signs that are not normal after getting Kanuma.
  • Tell your doctor if you are pregnant or plan on getting pregnant. You will need to talk about the benefits and risks of using Kanuma while you are pregnant.
  • Tell your doctor if you are breast-feeding. You will need to talk about any risks to your baby.

How is Kanuma best taken?

Use Kanuma as ordered by your doctor. Read all information given to you. Follow all instructions closely.

  • It is given as an infusion into a vein over a period of time.

What do I do if I miss a dose?

  • Call your doctor to find out what to do.

What are the side effects of Kanuma that I need to call my doctor about immediately?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

  • Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
  • Chest pain or pressure or a fast heartbeat.
  • Shortness of breath.
  • Eyelid swelling.
  • Runny nose.
  • Fast breathing.
  • Belly pain.
  • Feeling agitated.
  • Fever or chills.
  • Severe diarrhea.
  • Swelling.
  • Very bad headache.
  • Very bad dizziness or passing out.
  • Feeling irritable.
  • Very upset stomach or throwing up.
  • Pale skin.
  • Feeling very tired or weak.

What are some other side effects of Kanuma?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

  • Diarrhea.
  • Upset stomach or throwing up.
  • Cough.
  • Nose and throat irritation.
  • Headache.
  • Constipation.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out Kanuma?

  • If you need to store Kanuma at home, talk with your doctor, nurse, or pharmacist about how to store it.




  • NDC 25682-007-01
    Rx only
  • Kanuma®
    (sebelipase alfa)
  • 20 mg/10 mL
    (2 mg/mL)
  • For Intravenous Infusion Only
    Dilute Before Use
    Single-Use Only
    Discard Unused Portion
  • 1 vial



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