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news.The European Commission has granted marketing authorization to Shire for the use of Elaprase as the first ever enzyme replacement treatment for patients with Hunter syndrome.
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Elaprase replaces the enzyme, iduronate-2-sulfatase (I2S), that people with this disorder fail to produce in sufficient quantities. 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.
In a study treatment with Elaprase leads to improved endurance as measured by the six minute walk test. The trial also showed significant improvement in spleen and liver size and urine GAG levels.
Hunter syndrome is a very rare, progressive and life-threatening condition, which primarily affects males. The life expectancy for the more severely affected is only 10-20 years.
“This therapy offers the first real hope of long-term treatment for patients with Hunter syndrome, offering them the chance for an improvement in their symptoms and so their day-to-day life,” said Christine Lavery from MPS Society.
Marketing authorization for Elaprase follows positive opinion issued by The Committee for Medicinal Products for Human Use in October 2006. Elaprase will be launched across Europe over the next 18 months.
Shire gained Elaprase last year with its $1.3 billion acquisition of Transkaryotic Therapies, a biotechnology company that specialized in rare-disease drugs.