FDA OKS TREATMENT FOR HUNTER SYNDROME

The FDA has approved Elaprase (idursulfase), the first product for the treatment of Hunter syndrome, a rare inherited disease that can lead to premature death.

Hunter Syndrome, which usually becomes apparent in children 1 to 3 years of age, is a disease in which the person's body is defective in producing the chemical iduronate-2-sulfatase. Symptoms include growth delay, joint stiffness and coarsening of facial features. In severe cases, patients experience respiratory and cardiac problems, enlargement of the liver and spleen, neurological deficits and death. Hunter syndrome is diagnosed in approximately one out of 65,000 to 132,000 births.

The FDA had designated Elaprase as an orphan drug. Orphan products are generally developed to treat rare diseases or conditions that affect fewer than 200,000 people in the U.S. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first sponsor who obtains marketing approval for a designated orphan product.