Elaprase Approved for Rare Genetic Disorder

MONDAY, July 24, 2006 (HealthDay News) -- Elaprase (idursulfase) was approved Monday by the U.S. Food and Drug Administration as the first drug to treat Hunter syndrome, a rare, inherited disease that can cause premature death.

People with Hunter syndrome, which affects about one of every 65,000 to 132,000 births, cannot break down the body's complex sugars. Symptoms include stunted growth, joint stiffness, and coarse facial features. More severe effects include respiratory, cardiac and neurological problems, enlargement of the liver and spleen, and death, the FDA said in a statement.

Elaprase was approved as an orphan drug, meaning it was developed to treat a condition that affects fewer than 200,000 people. Such approval gives the Massachusetts-based manufacturer, Shire Human Genetic Therapies Inc., seven years of exclusive marketing rights.

Approval was granted following a 96-patient study that found treated patients were able to walk an average of 38 yards more in six minutes than untreated participants, the FDA said.

But some patients did suffer severe hypersensitivity reactions, prompting the agency to advise that "appropriate medical support should be readily available when Elaprase is administered."

More information

To learn more about Hunter syndrome, visit the U.S. National Library of Medicine.