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Kanuma

sebelipase alfa
Used for Inherited Metabolic Diseases, Lysosomal Storage Disease
Used for Inherited Metabolic Diseases, Lysosomal Storage Disease

Kanuma (sebelipase alfa) is an enzyme replacement therapy. It's used to treat a rare, genetic condition called lysosomal acid lipase (LAL) deficiency in adults and children. Kanuma (sebelipase alfa) is given as an infusion into the veins (IV) once every 1 to 2 weeks, depending on your age. Side effects of this medication can include fever, fever, and nausea.

Reviewed by:
reviewer
Robert Chad Hakim, PharmD, BCCCP
reviewer
Allison Gourley, PharmD
Last reviewed on November 15, 2023
BasicsSide effectsPros & consPharmacist tipsFAQsWarningsDosageReferencesPricesMedicare
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What is Kanuma (sebelipase alfa)?

What is Kanuma (sebelipase alfa) used for?

How Kanuma (sebelipase alfa) works

Kanuma (sebelipase alfa) is an enzyme replacement therapy for people with lysosomal acid lipase deficiency (LAL-D). People with LAL-D are missing an enzyme (protein) called lysosomal acid lipase (LAL), which is important for helping break down fats. Without it, fat builds up in your body, which can cause problems in many organs, like the liver and heart.

Kanuma (sebelipase alfa) replaces the missing LAL to help your body breakdown, absorb, and use fats.

Drug Facts

Common BrandsKanuma
Drug ClassEnzyme replacement therapy
Controlled Substance ClassificationNot a controlled medication
Generic StatusNo lower-cost generic available
AvailabilityPrescription only
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What are the side effects of Kanuma (sebelipase alfa)?

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Infants younger than 6 months old:
  • Diarrhea (67%)
  • Vomiting (67%)
  • Fever (56%)
  • Runny nose (56%)
  • Low red blood cell count (44%)
  • Cough (33%)
  • Sore throat, runny nose, and other signs of a common cold (33%)
  • Hives (33%)
Children and adults:
  • Headache (28%)
  • Fever (25%)
  • Pain in mouth and throat (17%)
  • Sore throat, runny nose, and other signs of a common cold (11%)
  • Lack of energy (8%)
  • Constipation (8%)
  • Nausea (8%)

Other Side Effects

Infants younger than 6 months old:
  • Weak muscle tone
  • Retching (dry heaving)
  • Sneezing
  • Fast heart rate
Children and adults:
  • Anxiety
  • Chest discomfort

Serious Side Effects

Contact your healthcare provider immediately if you experience any of the following.
  • Serious allergic reactions: pale skin, hives, rash, blisters, swelling of the lips or tongue, difficulty breathing, chest discomfort, nausea, vomiting, diarrhea, fever, chills, stomach pain, feeling irritated

Source: DailyMed

The following side effects have also been reported

Side effects that you should report to your care team as soon as possible:

Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):

pros-and-cons

Pros and cons of Kanuma (sebelipase alfa)

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Pros

Works well to treat LAL deficiency

Infusions given once every 1 to 2 weeks, depending on your age

Can be given to infants as young as 1 month old

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Cons

Needs to be given by a healthcare professional

Infusions typically last about 2 hours

Might not be a good option foe people with egg allergies

pharmacist-tips

Pharmacist tips for Kanuma (sebelipase alfa)

pharmacist

  • Each Kanuma (sebelipase alfa) appointment can take a while. The Kanuma (sebelipase alfa) infusion can last around 2 hours. After the infusion, your provider might ask you to stay so they can check your blood pressure, heart rate, and temperature. Please plan ahead for this time.

    • Kanuma (sebelipase alfa) can cause very serious allergic reactions, for up to 4 hours after your infusion. Get help right away if you experience signs of an allergic reaction, including stomach pain, fever, chills, diarrhea, nausea, swelling, itching, rash, and feeling agitated.

      • Let your provider know if you had an allergic reaction with Kanuma (sebelipase alfa) before. Depending on how serious your reaction was, your provider might stop this medication, give the infusion more slowly, or give you medications, like antihistamines or fever-reducers, before your future infusions.

        • Make sure to mark your appointments in your calendar to see your provider and receive your doses of Kanuma (sebelipase alfa). Missing infusions might make Kanuma (sebelipase alfa) work less well for you.

          • If your weight changes, let your provider know. Your dose of Kanuma (sebelipase alfa) depends on your weight.

            faqs

            Frequently asked questions about Kanuma (sebelipase alfa)

            What is lysosomal acid lipase deficiency (LAL-D)?
            Lysosomal acid lipase deficiency (LAL-D) is a rare, genetic problem that causes a buildup of fat throughout your body. This happens because the body can't make enough of an enzyme (protein) called LAL to help you break down fats. Early signs of LAL-D in young babies include poor weight gain, yellowing of the skin and whites of the eyes, vomiting, and diarrhea. Later on in life, LAL-D can damage organs like the heart, liver, spleen, and stomach. This can lead to issues such as a heart attack, stomach bleed, and liver problems.
            Is Kanuma (sebelipase alfa) a cure for lysosomal acid lipase deficiency?
            No, Kanuma (sebelipase alfa) doesn't cure LAL-D, it just treats the symptoms. LAL-D happens when the gene that makes LAL doesn't work properly. Kanuma (sebelipase alfa) is used to replace the missing LAL to help your body breakdown fats, but it doesn't change your genes. Since LAL-D is a genetic problem, you'll likely need to take Kanuma (sebelipase alfa) for your entire life. Your provider will have you continue taking the medication as long as it's helping your condition and you're not having severe side effects. Let your provider know if you have any questions or concerns about your treatment.
            Is Kanuma (sebelipase alfa) an orphan drug?
            Yes, Kanuma (sebelipase alfa) is an example of an orphan drug because it treats a rare medical condition.
            How long does each Kanuma (sebelipase alfa) infusion take?
            Your infusion time will depend on your dose of Kanuma (sebelipase alfa) and if you're having any reactions from the infusion. When you first start, your provider will infuse this medication over 2 hours or longer. If you tolerate it fine, they'll speed up your infusion in the future. But if you have a reaction to the infusion, such as stomach pain, agitation, fever, chills, and rash, your provider will likely slow down future infusions if you continue Kanuma (sebelipase alfa) treatment.
            Can I use Kanuma (sebelipase alfa) if I'm pregnant?
            There haven't been enough studies looking at the safety of Kanuma (sebelipase alfa) in pregnancy. Animal studies show babies weren't harmed when born to mothers receiving Kanuma (sebelipase alfa) doses much higher than the typical dose used in humans. If you're pregnant or thinking of getting pregnant, talk to your provider to see if Kanuma (sebelipase alfa) is right for you.
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            What are the risks and warnings for Kanuma (sebelipase alfa)?

            Kanuma (sebelipase alfa) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

            risk-warning

            Severe allergic reactions

            • Risk factors: Allergy to eggs or egg products

              Kanuma (sebelipase alfa) can cause serious, and sometimes life-threatening allergic reactions (e.g., anaphylaxis), typically within 4 hours of your infusion. These reactions have been reported as early as after the sixth infusion, to as late as a year after treatment. To prevent or ease reactions, your provider might infuse Kanuma (sebelipase alfa) at a slower rate or give you medications like antihistamines or acetaminophen (Tylenol) before your infusion. If you have a reaction that's not too severe, you might still be able to continue future treatments with Kanuma (sebelipase alfa). If you notice signs of an allergic reaction such as dizziness, a choking sensation, nausea, swelling around your eyes, swelling of the lips or tongue, or difficulty breathing, get medical attention right away.

              dosage

              Kanuma (sebelipase alfa) dosage forms

              Typical dosing for Kanuma (sebelipase alfa)

              Your provider will determine the dose based on your body weight.

              • Infants (ages under 6 months old): The typical starting dose is 1 mg/kg infused through the veins (IV) once per week. If needed, your child's provider will raise the dose to 3 mg/kg to 5 mg/kg once per week.

              • Adults and children (ages 6 months and older): The typical starting dose is 1 mg/kg IV once every 2 weeks. If needed, your provider will raise the dose to 3 mg/kg once every 2 weeks.

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              References

              Best studies we found

              Alexion Pharmaceuticals, Inc. (n.d.). Signs and symptoms of LAL-D (Lysosomal acid lipase deficiency).

              Alexion Pharmaceuticals, Inc. (n.d.). What is LAL-D? (Lysosomal acid lipase deficiency).

              Alexion Pharmaceuticals, Inc. (2021). Kanuma- sebelipase alfa injection, solution, concentrate [package insert]. DailyMed.

              View All References (5)

              American Liver Foundation. (n.d.). Lysosomal acid lipase deficiency (LALD).

              MedlinePlus. (2017). Lysosomal acid lipase deficiency.

              National Human Genome Research Institute. (2024). Gene.

              National Organization for Rare Disorders. (n.d.). Lysosomal acid lipase deficiency.

              Search Orphan Drug Designations and Approvals. (n.d.). Sebelipase alfa. U.S. Food and Drug Administration.

              GoodRx Health has strict sourcing policies and relies on primary sources such as medical organizations, governmental agencies, academic institutions, and peer-reviewed scientific journals. Learn more about how we ensure our content is accurate, thorough, and unbiased by reading our editorial guidelines.
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