idursulfase at a glance
Medicine class
Enzyme replacement therapy
Main use
Treatment of Hunter syndrome
Administration
Intravenous infusion under specialist supervision
Important risk
Infusion reactions, including rare life-threatening hypersensitivity
What is idursulfase used for?
Licensed use:
Treatment of Hunter syndrome, also called mucopolysaccharidosis II.
How does idursulfase work?
Hunter syndrome results from deficient iduronate-2-sulfatase, causing dermatan sulfate and heparan sulfate to accumulate within lysosomes. Idursulfase supplies a recombinant form of the enzyme, which cells take into lysosomes to break down accumulated glycosaminoglycans.
idursulfase preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 2 mg/mL
How to take idursulfase
Idursulfase is diluted and infused into a vein by trained healthcare professionals. People with severe airway disease require particularly careful monitoring. Home infusion may be considered only after treatment has been tolerated in a clinic and must remain medically supervised.
Tell the infusion team about fever, a respiratory infection or worsening breathing before treatment.
Report symptoms arising during the infusion immediately.
Do not prepare, mix or administer the concentrate yourself unless this is part of a formally supervised service.
idursulfase side effects
Common or expected effects
- Infusion-related reaction
- Headache or fever
- Flushing
- Rash, itching, hives or skin redness
- Wheezing or shortness of breath
- Nausea, vomiting, diarrhoea or abdominal pain
- Chest pain
Get urgent medical advice
- Anaphylactic or anaphylactoid reaction
- Severe breathing difficulty, bronchospasm or low oxygen levels
- Swelling of the tongue or face
- Marked blood-pressure changes, abnormal heart rhythm or blue skin
idursulfase in pregnancy
Human pregnancy information is very limited. The product information advises avoiding idursulfase during pregnancy as a precaution. Anyone pregnant or planning pregnancy should discuss the benefits and uncertainties with their specialist team.
idursulfase while breastfeeding
It is unknown whether idursulfase enters human milk, and risk to an infant cannot be excluded. Limited independent evidence suggests milk transfer and infant absorption may be low because idursulfase is a large protein. Breastfeeding and treatment should be reviewed with the specialist team.
idursulfase interactions
Formal interaction studies have not been performed. Clinically important cytochrome P450 interactions are not expected because idursulfase is processed within lysosomes. Give the infusion team a complete medicines list.
Common questions about idursulfase
Answers are fully visible for fast scanning and source review.
Is idursulfase a cure for Hunter syndrome?
No. It replaces the deficient enzyme and can reduce glycosaminoglycan accumulation, but does not correct the underlying genetic change.
Does it treat the neurological effects of Hunter syndrome?
Clinical evidence has not demonstrated benefit for neurological manifestations, partly because intravenously administered enzyme has limited access to the central nervous system.
Can infusions be given at home?
Home infusion may be considered after treatment has been well tolerated in a clinic, but it must remain supervised by an appropriate healthcare professional.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.