New biologic approved for Hunter syndrome

July 31, 2006

Replacement enzyme idursulfase (Elaprase, Shire Human Genetic Therapies) has been approved by the FDA as the first treatment for Hunter syndrome (mucopolysaccharidosis II), a rare disease in which the body cannot produce iduronate-2-sulfatase. The enzyme is necessary to break down complex sugars produced in the body, and its deficiency can lead to growth delay, coarsening of facial features, and joint stiffness

New biologic approved for Hunter syndrome

Replacement enzyme idursulfase (Elaprase, Shire Human Genetic Therapies) has been approved by the FDA as the first treatment for Hunter syndrome (mucopolysaccharidosis II), a rare disease in which the body cannot produce iduronate-2-sulfatase. The enzyme is necessary to break down complex sugars produced in the body, and its deficiency can lead to growth delay, coarsening of facial features, and joint stiffness. According to the FDA, the once-weekly therapy led to an average increased walking distance of 38 yards more for the treated group than for those patients taking placebo. The product will include a boxed warning alerting health professionals to the potential for infusion-related hypersensitivity reactions. In clinical trials, 15% of patients developed infusion-related reactions that affected two of three critical body systems defined as the skin, respiratory, or cardiovascular system. Eleven of the reactions were considered severe.

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