Hunter Syndrome normally shows up in children between one to three years of age. It causes a person's body to be defective in producing the chemical iduronate-2-sulfatase, which is needed to adequately breakdown complex sugars produced in the body. 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 even death.
Elaprase was designated as an orphan product by the FDA. Orphan products, such as Elaprase, are generally developed to treat rare diseases or conditions that affect fewer than 200,000 people in the U.S. Hunter syndrome is diagnosed in approximately one out of 65,000 to 132,000 births.
Dr. Steven Galson, Director, Center for Drug Evaluation and Research says in a statement, "This is the first product that brings help to a very small group of seriously ill patients who have no other treatment option."
He adds, "This approval is a good example of how the Orphan products program can benefit the public health with urgently needed products that would otherwise not be commercially available."
