The use of Elaprase® (idursulfase) for the long-term treatment of Hunter Syndrome patients has been granted marketing authorization by the European Commission.
Marketed by Shire Human Genetics Therapies, idursulfase is the first and only enzyme replacement treatment Hunter syndrome (Mucopolysaccharidosis II) sufferers since the condition has been identified 100 years ago.
Idursulfase, which is delivered by weekly infusions, replaces the deficient or missing enzyme, iduronate-2-sulfatase (I2S), that people with this progressive and debilitating genetic disorder fail to produce in sufficient quantities[2].
This lack of enzyme interferes with the body's ability to break down and recycle complex carbohydrates, common within secretions and cellular connective tissues, called glycosaminoglycans (GAG).
The build up of GAG causes damage to the body's tissues, which can result in hearing loss, declining heart function, an enlarged liver and spleen and a limited range of motion and mobility as a consequence of joint stiffness [2-5].
Idursulafase treatment lead to improved endurance and significant improvement in spleen, liver size and GAG levels, as shown in clinical trials. [1, 7, 8]
Primarily affecting males, Hunter Syndrome is a very rare, progressive life-threatening condition.
Find more details (including complete list of references) from Shire plc press release.
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