sebelipase alfa at a glance
Medicine class
Enzyme replacement therapy
Licensed use
Lysosomal acid lipase deficiency in patients of all ages
Administration
Intravenous infusion after dilution
Treatment setting
Specialist-supervised treatment given by a trained healthcare professional able to manage emergencies
Important allergy consideration
Severe reactions can occur, and the product may contain traces of egg proteins
What is sebelipase alfa used for?
Sebelipase alfa is licensed as enzyme replacement therapy for patients of all ages with lysosomal acid lipase deficiency.
It includes treatment of the rapidly progressive infantile form, often called Wolman disease.
It is also licensed for children and adults with later-presenting lysosomal acid lipase deficiency.
How does sebelipase alfa work?
Sebelipase alfa replaces deficient lysosomal acid lipase. After cells take it into lysosomes, it breaks down stored cholesteryl esters and triglycerides into free cholesterol, glycerol and fatty acids, reducing harmful fat accumulation.
How to take sebelipase alfa
Sebelipase alfa is not swallowed or self-injected. A trained healthcare professional dilutes it and administers it through a vein, with facilities available to manage severe infusion reactions.
Tell the team about egg allergy, previous infusion reactions and all other medicines before treatment.
Report symptoms during or after the infusion immediately.
sebelipase alfa side effects
Common or expected effects
- Infusion or hypersensitivity reactions, including itching, hives, chills, nausea or eczema
- Dizziness
- Abdominal pain or diarrhoea
- Tiredness or fever
- Rash or an infusion-site reaction
- In infants, vomiting, eyelid swelling, breathing difficulty, and changes in heart rate or temperature have also been reported
Get urgent medical advice
- Anaphylaxis or another severe hypersensitivity reaction, including breathing difficulty, throat swelling, wheezing, chest discomfort, marked pallor, low oxygen or cardiovascular symptoms
- A severe infusion reaction requiring the infusion to be stopped and urgent medical treatment
sebelipase alfa in pregnancy
Human pregnancy data are absent or very limited. As a precaution, the product information advises that sebelipase alfa should preferably be avoided during pregnancy. Discuss pregnancy or plans to conceive with the specialist team.
sebelipase alfa while breastfeeding
It is unknown whether sebelipase alfa enters human milk. The specialist and patient should decide whether to stop breastfeeding or interrupt treatment after considering the benefits to the child and mother.
sebelipase alfa interactions
Formal interaction studies have not been performed. As a recombinant human protein, sebelipase alfa is considered unlikely to cause cytochrome P450-mediated interactions.
Common questions about sebelipase alfa
Answers are fully visible for fast scanning and source review.
What is sebelipase alfa used to treat?
It treats lysosomal acid lipase deficiency, an inherited condition that causes cholesteryl esters and triglycerides to accumulate within cells.
Is sebelipase alfa an enzyme replacement therapy?
Yes. It supplies a recombinant form of the lysosomal acid lipase enzyme that is missing or deficient.
Is sebelipase alfa used for Wolman disease?
Yes. Wolman disease is the rapidly progressive infantile form of lysosomal acid lipase deficiency and falls within the licensed indication.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.