FDA APPROVES SHIRE'S HUNTER SYNDROME TREATMENT

The FDA approved Shire Pharmaceuticals' Elaprase, the first treatment for Hunter syndrome, the agency announced July 24.

Elaprase (idursulfase) provides the only treatment option for the isease, a rare inherited disorder that can lead to an early death. The disease is diagnosed in approximately one out of between 65,000 and 132,000 births.

"This is the first product that brings help to a very small group of seriously ill patients who have no other treatment option," Steven Galson, director of the Center for Drug Evaluation and Research, said. "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."

With Hunter syndrome, a person's body is defective in producing the chemicals necessary to breakdown complex sugars. This can cause 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 deficiencies and death.

The drug, designated as an orphan product, was shown to help patients walk, the FDA said. Orphan products are generally developed to treat rare diseases or conditions that affect fewer than 200,000 people in the U.S. The designation provides the developer with such incentives as a waiver of filing fees, grants for clinical trials and seven years of marketing exclusivity.