laronidase at a glance
Medicine class
Recombinant lysosomal enzyme replacement therapy
Licensed use
Non-neurological manifestations of confirmed MPS I
Administration
Diluted and given by intravenous infusion under specialist supervision
Key safety issue
Infusion-associated and serious allergic reactions can occur
What is laronidase used for?
Laronidase is licensed as enzyme replacement therapy for confirmed MPS I caused by alpha-L-iduronidase deficiency.
It treats non-neurological manifestations of the disease.
Clinical benefit for neurological manifestations has not been demonstrated.
How does laronidase work?
In MPS I, dermatan sulfate and heparan sulfate accumulate because alpha-L-iduronidase is deficient. Laronidase supplies a recombinant version of this enzyme. Cells take it into lysosomes, where it helps break down these stored substances and limit further accumulation.
How to take laronidase
Healthcare professionals dilute the concentrate in sodium chloride solution before intravenous infusion. An inherited-metabolic-disease specialist should supervise treatment, with facilities available to manage serious reactions.
Home infusion may be considered only after specialist assessment for people who have tolerated treatment well.
A trained healthcare professional must supervise home infusions and remain available afterwards.
Do not alter infusion settings without healthcare-professional supervision.
laronidase side effects
Common or expected effects
- Headache, nausea or abdominal pain
- Rash, flushing or itching
- Joint, back, muscle or limb pain
- Fever, chills or flu-like symptoms
- Infusion-site pain, redness, swelling or discomfort
- Dizziness, cough, vomiting or diarrhoea
- Changes in heart rate, blood pressure or oxygen saturation during an infusion
Get urgent medical advice
- Anaphylaxis or another severe hypersensitivity reaction
- Severe breathing difficulty, bronchospasm or respiratory failure
- Throat, face, lip or tongue swelling
- Airway obstruction, stridor, blue skin or markedly reduced oxygen levels
- Severe low blood pressure, fainting or collapse
laronidase in pregnancy
Human pregnancy data are inadequate. UK product information advises use only when clearly necessary after specialist assessment. One monitored pregnancy had a favourable outcome, but a single report cannot establish safety.
laronidase while breastfeeding
UK product information recommends stopping breastfeeding during treatment because infant-exposure data are insufficient. One case found no detectable laronidase in breast milk and no reported infant harm, but this limited evidence does not replace specialist advice.
laronidase interactions
Formal interaction studies are limited. Tell the specialist team about all prescribed, non-prescribed and complementary products.
Chloroquine
Do not administer simultaneously because it may interfere with laronidase uptake into cells.
Procaine
Do not administer simultaneously because it may interfere with laronidase uptake into cells.
Common questions about laronidase
Answers are fully visible for fast scanning and source review.
What is laronidase used for?
It treats non-neurological manifestations of confirmed MPS I by replacing deficient alpha-L-iduronidase.
Does it treat brain and nerve symptoms of MPS I?
Clinical benefit for neurological manifestations has not been demonstrated.
Can it be given at home?
Possibly, after specialist assessment, if treatment has been well tolerated and trained healthcare support and emergency procedures are available.
Why might medicines be given beforehand?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.