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Treatment for Mucopolysaccharidosis Type II

Update: Elaprase Now FDA Approved - July 24, 2006

Shire Files Elaprase (idursulfase) With the FDA for the Treatment of Hunter Syndrome

Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare, life threatening, genetic disorder with no available treatment. Individuals with Hunter syndrome lack the enzyme iduronate-2-sulfatase, which is essential in the continuous process of replacing and breaking down glycosaminoglycans (GAG). As a result, GAG remains stored in cells in the body causing progressive damage. The symptoms of Hunter syndrome are usually not visible at birth, but usually start to become noticeable after the first or second year of life. Often the first symptoms may include hernias, frequent ear infections, runny noses, reduced growth rate and abnormal facial appearance.

As the disease progresses, a variety of symptoms appear including enlarged liver and spleen, heart failure, decreased endurance, obstructive and restrictive airway disease, sleep apnea, joint stiffness, and, in some cases, central nervous system involvement. If central nervous system involvement exists, the life expectancy for patients with Hunter syndrome is typically 10-15 years of age, however, some patients can survive into the fifth or sixth decade of life. There is currently no effective therapy for Hunter syndrome.

Elaprase is a human iduronate-2-sulfatase produced by genetic engineering technology, developed to replace the missing enzyme in Hunter syndrome patients. Elaprase has been designated an orphan drug in both the United States and in the European Union.

Source: Shire Pharmaceuticals Group plc

Posted: November 2005

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