Sebelipase alfa parenteral
- Drugs List
- Therapeutic Indications
- Dosage
- Administration
- Contraindications
- Precautions and Warnings
- Pregnancy and Lactation
- Side Effects
- Monograph
Presentation
Solution for infusion containing sebelipase alfa.
These products have been produced by recombinant DNA technology in egg white of transgenic Gallus.
Drugs List
Therapeutic Indications
Uses
Lysosomal acid lipase deficiency
Dosage
Adults
1mg/kg once every other week.
Dose escalation to 3mg/kg once every other week should be considered based on suboptimal clinical response.
Children
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.
Administration
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.
Contraindications
Breastfeeding
Precautions and Warnings
Infants with multiple organ failure
Restricted sodium intake
Pregnancy
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
Pregnancy
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.
Lactation
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
Agitation
Anaphylaxis
Antibody formation
Bronchospasm
Chest discomfort
Chills
Conjunctival hyperaemia
Diarrhoea
Dizziness
Dyspnoea
Eczema
Extravasation
Eyelid oedema
Fatigue
Flushing
Hyperaemia
Hypercholesterolaemia
Hypersensitivity reactions
Hypertension
Hyperthermia
Hypertriglyceridaemia
Hypotension
Hypoxia
Increased heart rate
Increased respiratory rate
Infusion site reaction
Irritability
Laryngeal oedema
Maculopapular rash
Nausea
Oedema
Oxygen saturation decreased
Pallor
Pruritus
Pyrexia
Rash
Respiratory distress
Rhinorrhoea
Rise in body temperature
Stridor
Tachycardia
Urticaria
Vomiting
Overdosage
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 ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).
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.
Medscape UK | Univadis prescription drug monographs & interactions are based on FDB Multilex Content
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