This site is intended for UK healthcare professionals
Medscape UK Univadis Logo
Medscape UK Univadis Logo

Sebelipase alfa parenteral


Solution for infusion containing sebelipase alfa.

These products have been produced by recombinant DNA technology in egg white of transgenic Gallus.

Drugs List

  • KANUMA 20mg/10ml concentrate for solution for infusion
  • sebelipase alfa 20mg/10ml concentrate for solution for infusion
  • Therapeutic Indications


    Lysosomal acid lipase deficiency



    1mg/kg once every other week.

    Dose escalation to 3mg/kg once every other week should be considered based on suboptimal clinical response.


    Children aged 6 months to 18 years who do not present with rapidly progressive lysosomal acid lipase (LAL) deficiency
    1mg/kg once every other week.

    Dose escalation to 3mg/kg once every other week should be considered based on suboptimal clinical response.

    Infants under 6 months of age with rapidly progressive LAL deficiency
    1mg/kg or 3mg/kg once weekly. The higher starting dose should be based on the severity of the disease and rapid disease progression.

    Dose escalation to 3mg/kg once weekly to be considered if suboptimal clinical response after a minimum of 4 infusions and a further dose escalation up to 5mg/kg once weekly can be considered if persistent suboptimal clinical response.

    Further dose adjustments, as a reduction of the dose or an extension of the dose interval can be made on an individual basis. Clinical studies evaluated doses ranging from 0.35mg/kg to 5mg/kg once weekly, with one patient receiving a higher dose of 7.5mg/kg once weekly. Doses higher than 7.5mg/kg once weekly have not been studied.


    To be administered as an intravenous infusion.

    It is important to initiate treatment as early as possible after diagnosis of LAL deficiency.

    The total volume of the infusion should be administered over approximately 2 hours. A 1 hour infusion may be considered after patient tolerability is established. The infusion period may be extended in the event of dose escalation.



    Precautions and Warnings

    Infants with multiple organ failure
    Restricted sodium intake

    Contains more than 1 mmol (23 mg) sodium per dose
    Advise patient dizziness may affect ability to drive or operate machinery
    Consider pre-medication with antihistamines and/or antipyretics
    Consider use of corticosteroids if adverse reactions occur
    Treatment to be initiated and supervised by a specialist
    May contain trace amounts of egg phospholipids
    If adverse reactions occur, reduce rate or temporarily stop infusion
    Record name and batch number of administered product
    Resuscitation facilities must be immediately available
    Slow infusion and treat non life threatening allergic reactions accordingly
    Antibodies to ingredient may develop
    Monitor for hypersensitivity reactions for 1 hour after administration
    Discontinue if serious allergic or anaphylactic reaction occurs

    Following the first infusion of sebelipase alfa, including the first infusion after a dose escalation, patients should be observed for 1 hour in order to monitor for any signs or symptoms of anaphylaxis or a severe hypersensitivity reaction.

    If severe reactions occur, sebelipase alfa infusion should be stopped and appropriate medical treatment should be initiated immediately. It is important to be cautious and consider the risks and benefits of re-administering sebelipase alfa following a severe reaction.

    Patients should be tested for the presence of antibodies in cases of severe infusion reactions and in cases of lack or loss of effect.

    Pregnancy and Lactation


    Use sebelipase alfa with caution during pregnancy.

    The manufacturer advises as a precautionary measure that it is preferable to avoid use of sebelipase alfa during pregnancy. At the time of writing there is limited published information regarding the use of sebelipase alfa during pregnancy. Animal studies do not indicate direct or indirect harmful effects with respect to reproductive toxicity.


    Sebelipase alfa is contraindicated during breastfeeding.

    The manufacturer recommends that a decision must be made whether to discontinue breastfeeding or to discontinue sebelipase alfa therapy taking into account the benefit of breastfeeding for the child and the benefit of therapy for the woman. At the time of writing there is limited published information regarding the use of sebelipase alfa during breastfeeding. It is not known whether sebelipase alfa is excreted in human milk.

    Side Effects

    Abdominal distension
    Abdominal pain
    Antibody formation
    Chest discomfort
    Conjunctival hyperaemia
    Eyelid oedema
    Hypersensitivity reactions
    Increased heart rate
    Increased respiratory rate
    Infusion site reaction
    Laryngeal oedema
    Maculopapular rash
    Oxygen saturation decreased
    Respiratory distress
    Rise in body temperature


    It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

    The following number will direct the caller to the relevant local centre (0844) 892 0111

    Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( ) or if this is unavailable at the backup site ( ).

    Further Information

    Last Full Review Date: June 2020

    Reference Sources

    Summary of Product Characteristics: Kanuma 2mg/ml concentrate solution. Alexion Pharma UK Ltd. Revised February 2022.

    Access the full UK drug database with a FREE Medscape UK Account
    It takes just a few minutes, and you’ll get unlimited access to information on over 11,000 UK drugs.
    Register for Free

    Already a member? Log in

    Medscape UK | Univadis prescription drug monographs & interactions are based on FDB Multilex Content

    FDB Logo

    FDB Disclaimer : FDB Multilex is intended for the use of healthcare professionals and is provided on the basis that the healthcare professionals will retain FULL and SOLE responsibility for deciding what treatment to prescribe or dispense for any particular patient or circumstance.