Sebelipase alfa treats those with lysosomal acid lipase deficiency, an enzyme disorder. This is the first treatment for this rare disease.
Sebelipase alfa Overview
Sebelipase alfa is a prescription medication used to treat the rare enzyme disorder lysosomal acid lipase (LAL) deficiency. Sebelipase alfa belongs to a group of drugs called human lysosomal acid lipase (rhLAL) proteins. These work by providing a rhLAL protein that functions in place of the missing, partially active or inactive LAL protein in patients.
This medication is available in an injectable form to be given directly into a vein (IV) by a healthcare professional.
Common side effects of sebelipase alfa include diarrhea, vomiting, and fever.
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Uses of Sebelipase alfa
Sebelipase alfa is a prescription medication used to treat children and adults with lysosomal acid lipase (LAL) deficiency. LAL deficiency is also known as Wolman disease and cholesteryl ester storage disease (CESD). People with these conditions have little or no LAL enzyme activity, which can cause a build-up of fats within cells of different tissues that can lead to liver and heart disease, as well as other health problems.
This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.
Sebelipase alfa Brand Names
Sebelipase alfa may be found in some form under the following brand names:
Sebelipase alfa Drug Class
Sebelipase alfa is part of the drug class:
Side Effects of Sebelipase alfa
Serious side effects have been reported with sebelipase alfa. See the "Drug Precautions" section.
Common side effects include the following:
- rhinitis (symptoms affecting the nose)
- anemia (low iron in blood)
This is not a complete list of sebelipase alfa side effects. Ask your doctor or pharmacist for more information.
Tell your doctor if you have any side effect that bothers you or that does not go away.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
Sebelipase alfa Interactions
No sebelipase alfa drug interactions have been reported by the manufacturer. However, you should tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. Not all drug interactions are known or reported and new drug interactions are continually being reported.
Sebelipase alfa Precautions
Serious side effects have been reported with sebelipase alfa including the following:
Hypersensitivity reactions including anaphylaxis (severe allergic reaction). Your doctor will monitor you during your infusion for signs of a hypersensitivity reaction. Tell your healthcare provider about any signs or symptoms of hypersensitivity, which include the following:
- chest pain
- swelling of the face, eyes, lips, tongue, arms, or legs
- difficulty breathing or swallowing
- fast heartbeat
- stomach pain
- fever or chills
- nausea or vomiting
- Hypersensitivity to eggs or egg products. Sebelipase alfa is made in the egg whites of genetically engineered chickens. Patients with a known allergy to eggs or egg products will need to talk to their healthcare provider about the risks and benefits of using sebelipase alfa before starting treatment.
Do not take sebelipase alfa if you are allergic to sebelipase alfa or to any of its ingredients.
Sebelipase alfa Food Interactions
Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of sebelipase alfa, there are no specific foods that you must exclude from your diet when receiving this medication.
Before taking sebelipase alfa, tell your doctor about all of your medical conditions. Especially tell you doctor if you:
- are allergic to sebelipase alfa or to any of its ingredients
- have a known allergy to egg or egg products
- are pregnant or plan to become pregnant
- are breastfeeding or plan to breastfeed
Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.
Sebelipase alfa and Pregnancy
Tell your doctor if you are pregnant or plan to become pregnant.
There are no studies that have been done in humans with sebelipase alfa. In animal studies, pregnant animals were given this medication, and the babies did not show any medical issues related to this medication. Talk to your healthcare provider about the risks and benefits of taking sebelipase alfa while pregnant.
Sebelipase alfa and Lactation
Tell your doctor if you are breastfeeding or plan to breastfeed.
It is not known if sebelipase alfa crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using sebelipase alfa.
Sebelipase alfa Usage
Use sebelipase alfa exactly as prescribed.
Sebelipase alfa comes in injectable form to be given directly into a vein (IV) by a healthcare provider, either once a week or once every 2 weeks. The infusion is usually given over at least 2 hours.
Your doctor may give you medications to help prevent a reaction to the infusion, especially if you have had a reaction before.
If you miss an appointment for a dose of sebelipase alfa, contact your healthcare provider as soon as possible to reschedule.
Sebelipase alfa Dosage
The dose your doctor recommends may be based on the following:
- your weight
- the condition being treated
- how you respond to this medication
Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life
The recommended dose of Kanuma (sebelipase alfa) for infants less than 6 months old and who have a quickly progressing disease is 1 mg/kg given once weekly. This dose can be increased to a maximum of 3 mg/kg weekly for patients who do not achieve the desired response at the lower dose.
Pediatric and Adult Patients with LAL Deficiency
The recommended dose for children and adults with LAL deficiency is 1 mg/kg given once every other week.
Sebelipase alfa Overdose
If sebelipase alfa is administered by a healthcare provider in a medical setting, it is unlikely that an overdose will occur. However, if overdose is suspected, seek emergency medical attention.
Make sure to keep all appointments with your doctor and the laboratory.