XML Feed for RxPG News   Add RxPG News Headlines to My Yahoo!   Javascript Syndication for RxPG News

Research Health World General
 Latest Research
  X Chromosome
  Genetic Disorders
   Fragile X Syndrome
 Infectious Diseases
 Respiratory Medicine
 Public Health
 Clinical Trials
 Medical News
 Awards & Prizes
 Special Topics
 Odd Medical News
 World News

Last Updated: Aug 19th, 2006 - 22:18:38

Genetic Disorders Channel
subscribe to Genetic Disorders newsletter

Latest Research : Genetics : Genetic Disorders

   DISCUSS   |   EMAIL   |   PRINT
FDA Approves Idursulfase As First Treatment for Hunter Syndrome
Aug 2, 2006, 12:30, Reviewed by: Dr. Priya Saxena

"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."

The Food and Drug Administration (FDA) approved Elaprase (idursulfase), the first product for the treatment of Hunter syndrome (Mucopolysaccharidosis II, or MPS II), a rare inherited disease which can lead to premature death. Elaprase is a new molecular entity, which is an active ingredient never before marketed in the United States.

Hunter Syndrome, which usually becomes apparent in children one to three years of age, is a disease in which the person's body is 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 death.

Elaprase was designated as an orphan product by 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. 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. Hunter syndrome is diagnosed in approximately one out of 65,000 to 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," said Dr. Steven Galson, Director, Center for Drug Evaluation and Research. "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."

Elaprase was approved after a randomized, double-blind, placebo-controlled study of 96 patients with Hunter syndrome showed that the treated participants had an improved capacity to walk. At the end of the 53–week trial, patients who received Elaprase infusions experienced on average a 38-yard greater increase in the distance walked in six minutes compared to the patients on placebo.

The most serious adverse events reported during the trial were hypersensitivity reactions to Elaprase that could be life-threatening. They included respiratory distress, drop in blood pressure, and seizure. Other frequent, but less serious adverse events included fever, headache and joint pain.

Because of the potential for severe hypersensitivity reactions, appropriate medical support should be readily available when Elaprase is administered. Patients and their physicians are encouraged to participate in a voluntary Hunter Outcome Survey which has been established to monitor and evaluate the safety and effects of long-term treatment with Elaprase.

- Food and Drug Administration (FDA), U.S.


Subscribe to Genetic Disorders Newsletter
E-mail Address:


Elaprase is manufactured by Shire Human Genetic Therapies, Inc., in Cambridge, MA.

Related Genetic Disorders News

Williams Syndrome, the brain and music
Exploring genetics of congenital malformations
FDA Approves Idursulfase As First Treatment for Hunter Syndrome
PARP1 inhibitors can protect Huntington's disease affected cells from damage
Gene therapy protects neurons in Huntington's disease
Huntingtin cleavage is caused by caspase-6
The risk of transmission of genetic disorders through donor's sperm
Liver transplants provide metabolic cure for maple syrup urine disease
Spinocerebellar ataxia type 5 (SCA5) gene pinpointed
Scientists probe connection between regulatory DNA and disease

For any corrections of factual information, to contact the editors or to send any medical news or health news press releases, use feedback form

Top of Page


© Copyright 2004 onwards by RxPG Medical Solutions Private Limited
Contact Us