RxPG News Feed for RxPG News

Medical Research Health Special Topics World
  Home
 
   Health
 Aging
 Asian Health
 Events
 Fitness
 Food & Nutrition
 Happiness
 Men's Health
 Mental Health
 Occupational Health
 Parenting
 Public Health
 Sleep Hygiene
 Women's Health
 
   Healthcare
 Africa
 Australia
 Canada Healthcare
 China Healthcare
 India Healthcare
 New Zealand
 South Africa
 UK
 USA
 World Healthcare
 
   Latest Research
 Aging
 Alternative Medicine
 Anaethesia
 Biochemistry
 Biotechnology
 Cancer
 Cardiology
 Clinical Trials
 Cytology
 Dental
 Dermatology
 Embryology
 Endocrinology
 ENT
 Environment
 Epidemiology
 Gastroenterology
 Genetics
  Cloning
  Genetic Disorders
   Brachydactyly
   Fragile X Syndrome
   Huntington's
   MSUD
   Progeria
  X Chromosome
 Gynaecology
 Haematology
 Immunology
 Infectious Diseases
 Medicine
 Metabolism
 Microbiology
 Musculoskeletal
 Nephrology
 Neurosciences
 Obstetrics
 Ophthalmology
 Orthopedics
 Paediatrics
 Pathology
 Pharmacology
 Physiology
 Physiotherapy
 Psychiatry
 Radiology
 Rheumatology
 Sports Medicine
 Surgery
 Toxicology
 Urology
 
   Medical News
 Awards & Prizes
 Epidemics
 Launch
 Opinion
 Professionals
 
   Special Topics
 Ethics
 Euthanasia
 Evolution
 Feature
 Odd Medical News
 Climate

Last Updated: Oct 11, 2012 - 10:22:56 PM
Genetic Disorders Channel

subscribe to Genetic Disorders newsletter
Latest Research : Genetics : Genetic Disorders

   EMAIL   |   PRINT
FDA Approves Idursulfase As First Treatment for Hunter Syndrome

Aug 2, 2006 - 12:30:00 PM , Reviewed by: 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."

 
[RxPG] 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.



Publication: Food and Drug Administration (FDA), U.S.
On the web: www.fda.gov 

Advertise in this space for $10 per month. Contact us today.


Related Genetic Disorders News
XXYY syndrome- features and treatment options elucidated by researchers
Switching genes to overdrive improves muscular dystrophy symptoms in mice
Gene mutation associated with X-linked mental retardation revealed
Link between Huntington's and abnormal cholesterol levels in brain discovered
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

Subscribe to Genetic Disorders Newsletter

Enter your email address:


 Additional information about the news article
Elaprase is manufactured by Shire Human Genetic Therapies, Inc., in Cambridge, MA.
 Feedback
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

 
Contact us

RxPG Online

Nerve

 

    Full Text RSS

© All rights reserved by RxPG Medical Solutions Private Limited (India)