Recombinant human lysosomal acid lipase enzyme replacement Pregnancy: Available data in pregnant women are insufficient to identify a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. Animal reproduction studies showed no evidence of embryolethality, fetotoxicity, teratogenicity or abnormal early embryonic development at exposures up to 164-fold (rats) and 526-fold (rabbits) the human dose of 1 mg/kg every other week.

Sebelipase alfa

Brand names: Kanuma

Sebelipase alfa is a recombinant human lysosomal acid lipase enzyme replacement therapy for lysosomal acid lipase deficiency.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: Adult and paediatric patients with lysosomal acid lipase (LAL) deficiency: 1 mg/kg administered as an intravenous infusion once every other week; for patients with a suboptimal clinical response, increase the dosage to 3 mg/kg once every other week

Route: Intravenous infusion only — dilute in 0.9% sodium chloride to a final concentration of 0.1 mg/mL to 1.5 mg/mL and administer using a low-protein binding infusion set with an in-line, low-protein binding 0.2 micron filter

Frequency: Once every other week

Max: 3 mg/kg once every other week in paediatric and adult patients with LAL deficiency (up to 5 mg/kg once weekly in infants with rapidly progressive LAL deficiency presenting within the first 6 months of life)

No UK SPC was present in the fetched source bundle (providers.emc was null) — this draft is taken from the US FDA prescribing information for KANUMA (Alexion, label date 2025-07-21) and must be verified against the UK SPC before publication. Infuse over at least 2 hours; consider further prolonging the infusion time for patients receiving dosages greater than 1 mg/kg or for those who have experienced a hypersensitivity reaction; a 1-hour infusion may be considered for the 1 mg/kg dose in patients who tolerate the infusion. Administration should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis, and initiated in a healthcare setting with appropriate medical monitoring and support measures including access to cardiopulmonary resuscitation equipment. A suboptimal clinical response is defined as poor growth, deteriorating biochemical markers (e.g. ALT, AST) and/or parameters of lipid metabolism (e.g. LDL-c, triglycerides). Supplied as 20 mg/10 mL (2 mg/mL) single-dose vials; total dose (mg) = weight (kg) x dose (mg/kg), total vials = total dose divided by 20 mg/vial (round up to the next whole vial). Contains no preservatives — use immediately after dilution; if not possible, store up to 24 hours at 2 to 8 degrees C, protected from light; do not freeze or shake.

Paediatric dose

Dose: 1 mg/kg

Route: Intravenous infusion over at least 2 hours (a 1-hour infusion may be considered for the 1 mg/kg dose in patients who tolerate it)

Frequency: Once every other week in paediatric patients with LAL deficiency; once weekly in infants with rapidly progressive LAL deficiency presenting within the first 6 months of life

Max: 3 mg/kg once every other week in paediatric patients with LAL deficiency; 5 mg/kg once weekly in infants with rapidly progressive LAL deficiency presenting within the first 6 months of life

From US FDA prescribing information for KANUMA — no UK SPC was available in the source bundle. Infants with rapidly progressive LAL deficiency presenting within the first 6 months of life: recommended starting dosage 1 mg/kg IV once weekly; for suboptimal clinical response increase to 3 mg/kg once weekly, and for continued suboptimal response on 3 mg/kg once weekly further increase to 5 mg/kg once weekly. Suboptimal clinical response is defined as poor growth, deteriorating biochemical markers or persistent or worsening organomegaly. Safety and effectiveness have been established in paediatric patients aged 1 month and older. Clinician to verify against the UK SPC and a children's formulary.

Dose auto-extracted from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Paediatric weight-based calculator

Patient weight (kg)

Verify in a children's formulary

Contraindications

None stated — the US label records "CONTRAINDICATIONS: None"

Side effects

Infants with rapidly progressive LAL deficiency presenting within the first 6 months of life (>= 30%): diarrhoea, vomiting, fever, rhinitis, anaemia, cough, nasopharyngitis, urticaria
Paediatric and adult patients with LAL deficiency (>= 8%): headache, fever, oropharyngeal pain, nasopharyngitis, asthenia, constipation, nausea
Hypersensitivity reactions, reported in 21 of 106 (20%) treated patients in clinical trials
Life-threatening hypersensitivity reactions including anaphylaxis (3 of 106 patients, all infants, in clinical trials)
Consider the risks and benefits of treatment in patients with known systemic hypersensitivity reactions to eggs or egg products

Clinical monograph

How it works

It replaces deficient lysosomal acid lipase, hydrolysing accumulated cholesteryl esters and triglycerides within lysosomes.

Prescribing in practice

Hypersensitivity and infusion-associated reactions, including anaphylaxis, can occur; administer under supervision with resuscitation facilities available.
It is given by intravenous infusion at regular intervals; refer to the SPC for administration details and the approach in rapidly progressive infantile disease.
Anti-drug antibodies may develop and can influence response and tolerability.

Monitoring

Patients are monitored during and after infusions for hypersensitivity, with review of lipid profile, hepatic parameters and antibody status as indicated.

Counselling the patient

This treatment is given as a regular drip and is a long-term therapy.
Report any rash, breathing difficulty, flushing or feeling unwell during or after an infusion.
Attend scheduled appointments for ongoing monitoring.

Evidence & guidelines

Approval was supported by clinical trials in infants, children and adults with lysosomal acid lipase deficiency.

Reference: NICE HST4; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

Calculators

Pathways