Kanuma
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.
What is Kanuma (sebelipase alfa)?
What is Kanuma (sebelipase alfa) used for?
- Lysosomal acid lipase deficiency (LAL-D) in adults and children ages 1 month and older
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 Brands | Kanuma |
|---|---|
| Drug Class | Enzyme replacement therapy |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
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%)
Less Common Side Effects
Infants younger than 6 months old:
- Weak muscle tone
- Retching (dry heaving)
- Sneezing
- Fast heart rate
Children and adults:
- Anxiety
- Chest discomfort
Kanuma (sebelipase alfa) 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
The following Kanuma (sebelipase alfa) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Infusion reactions—chest pain, shortness of breath or trouble breathing, feeling faint or lightheaded
Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):
- Cough
- Diarrhea
- Itching
- Runny or stuffy nose
- Sore throat
- Vomiting
Pros and cons of Kanuma (sebelipase alfa)
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
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 for Kanuma (sebelipase alfa)
- 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.
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.
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.
Kanuma (sebelipase alfa) dosage
Typical dosage 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.
