The Pharmaceutical Journal
Vol 278 No 7449 p479
28 April 2007

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First enzyme replacement to treat Hunter syndrome

Idursulfase, a new long-term treatment for patients with Hunter syndrome (mucopolysaccharidosis II), has been launched by Shire Pharmaceuticals. It is the first enzyme replacement treatment for people suffering from Hunter syndrome since the condition was identified 100 years ago.

Marketed as Elaprase, idursulfase is a purified form of iduronate-2-sulfatase lysosomal enzyme, and is produced by recombinant DNA technology in a human cell line. It should be given at a dose of 0.5mg/kg every week by intravenous infusion over three hours (the infusion time can be gradually reduced to one hour if no infusion-associated reactions occur).

The product has been authorised by the European Medicines Agency under “exceptional circumstances”, which means that, due to the rarity of the disease, it has not been possible to obtain complete information on it. The EMEA will review any new information every year and the summary of product characteristics will be updated as necessary.

Hunter syndrome is a rare, progressive and life-threatening condition, which primarily affects males. It is one of several hereditary, metabolic diseases known as lysosomal storage disorders.


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