Laronidase

What it is and how it works

Laronidase is a recombinant form of human alpha-L-iduronidase, the lysosomal enzyme deficient in mucopolysaccharidosis type I. It is taken up into lysosomes via mannose-6-phosphate receptors and catalyses the breakdown of the glycosaminoglycans dermatan sulfate and heparan sulfate, reducing their accumulation in tissues.

Indications

Long-term enzyme replacement therapy for the non-neurological manifestations of confirmed mucopolysaccharidosis type I (alpha-L-iduronidase deficiency).

Dosage and administration (summary)

100 units/kg by intravenous infusion once weekly, with a gradually increasing infusion rate, under supervision of a physician experienced in MPS I or metabolic disorders.

Contraindications

Severe hypersensitivity (anaphylactic reaction) to laronidase or excipients.

Warnings and precautions

Infusion-associated reactions, including severe anaphylaxis, may occur; premedication with antihistamines and/or antipyretics is often used and resuscitation facilities must be available. Patients with compromised respiratory function or acute febrile or respiratory illness are at higher risk of severe reactions. Most patients develop IgG antibodies; it does not cross the blood–brain barrier and is not expected to treat CNS manifestations.

Pregnancy

Use only if clearly necessary.

Storage and supply

Store at 2–8 °C; do not freeze; use diluted solution promptly.

Source: reference summary prepared from the EU SmPC/EPAR, BNF and WHO Model Formulary. The approved product information in Iran governs.