Navigation Links
FDA Approves Ferriprox to Treat Patients with Excess Iron in the Body
Date:10/14/2011

SILVER SPRING, Md., Oct. 14, 2011 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Ferriprox (deferiprone) to treat patients with iron overload due to blood transfusions in patients with thalassemia, a genetic blood disorder that causes anemia, who had an inadequate response to prior chelation therapy.

(Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO)

Patients with thalassemia have excess iron in the body from the frequent blood transfusions (transfusional iron overload), a condition that is serious and can be fatal.  These patients also have a risk of developing liver disease, diabetes, arthritis, heart failure or an abnormal heart rhythm.  

The standard of care to treat transfusional iron overload is chelation therapy – chemical agents that are used to remove heavy metals from the body. Ferriprox is intended for use when chelation therapy is inadequate.

"Ferriprox represents the first new FDA-approved treatment for this disorder since 2005," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.

The safety and effectiveness of Ferriprox is based on an analysis of data from twelve clinical studies in 236 patients. Patients participating in the study did not respond to prior iron chelation therapy.  Ferriprox was considered a successful treatment for patients who experienced at least a 20 percent decrease in serum ferritin, a protein that stores iron in the body for later use. Half of the patients in the study experienced at least a 20 percent decrease in ferritin levels.

The most common side effects seen in patients who received Ferriprox included nausea, vomiting, abdominal and joint pain, urine discoloration (chromaturia), a decrease in the number of white blood cells (neutropenia), and an increase in the level of a liver enzyme that may be indicative of tissue or liver damage at unsafe amounts.

The most serious side effect seen in about two percent of patients treated with Ferriprox was the development of agranulocytosis, a serious and potentially life-threatening reduction in the number of granulocytes (a type of white blood cell that fights infection).

The therapy is being approved under the FDA's accelerated approval program, designed to provide patients with earlier access to promising new drugs followed by further studies to confirm the drug's clinical benefit.

The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than survival or irreversible morbidity (illness).

ApoPharma has agreed to several post-marketing requirement and commitments. One commitment includes further study of the use of Ferriprox in patients with sickle cell disease who have transfusional iron overload.

Earlier this year, the U.S. Department of Health and Human Services (HHS) launched the Sickle Cell Disease (SCD) Initiative bringing together HHS agencies to enhance the quality and quantity of SCD data, develop best practice guidelines and quality of care metrics, improve health care delivery and coordination of care for patients with SCD, facilitate approval of new medical products, and expand research on SCD.  The post-marketing requirement for further study of Ferriprox aligns with the goals of the SCD Initiative.

Ferriprox is marketed by ApoPharma Inc. of Toronto.

For more information:

FDA: Office of Hematology and Oncology Products
http://www.fda.gov/AboutFDA/CentersOffices/CDER/ucm091745.htm

FDA: Spotlight on Drug Innovation – Update of FDA's novel drug approvals in 2011
http://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm254242.htm

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Media Inquiries: Erica Jefferson, 301-796-4988, erica.jefferson@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA


'/>"/>
SOURCE U.S. Food and Drug Administration
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. FDA Approves Lillys Osteoporosis Drug EVISTA(R) (raloxifene HCl) to Reduce The Risk of Invasive Breast Cancer in Two Populations of Postmenopausal Women
2. Health Canada Approves ATRIPLA(R) (efavirenz 600 mg/emtricitabine 200 mg/ tenofovir disoproxil fumarate 300 mg), the First Once-Daily Single Tablet Regimen for HIV
3. FDA Approves Cymbalta(R) for Maintenance Treatment of Major Depressive Disorder
4. European Commission Approves ATRIPLA(R) (efavirenz 600 mg/emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg), the First Once-Daily Single Tablet Regimen for Virologically Suppressed Adults With HIV-1 Infection
5. FDA Approves MiddleBrooks Amoxicillin PULSYS for Pharyngitis/Tonsillitis in Adolescents and Adults
6. FDA Approves XYZAL(R) (levocetirizine dihydrochloride) Oral Solution for the Relief of Seasonal and Year Round Allergies and Chronic Idiopathic Urticaria
7. UM Maryland Industrial Partnerships Program Approves 18 Collaborative Research Projects, Worth $4.4 Million, Between University Faculty, State Technology Companies
8. U.S. Food and Drug Administration Approves ABILIFY(R) (aripiprazole) for the Acute Treatment of Manic and Mixed Episodes Associated With Bipolar I Disorder in Pediatric Patients (10 to 17 Years of Age)
9. U.S. Food and Drug Administration Approves ORENCIA(R) (abatacept) for the Treatment of Moderate-to-Severe Polyarticular Juvenile Idiopathic Arthritis (JIA) in Patients Six Years and Older
10. FDA Approves HIFU Clinical Trial for Recurrent Prostate Cancer
11. EPA Approves XDS-CALUX(R) as Alternate Dioxin Test Method 4435
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/28/2016)... , Net Sales of $1.90 billion represent an increase of ... and an increase of 1.2% on an adjusted pro forma, ... quarter were $0.52 reported, a decrease of 47.5% from the ... over the prior year period , The Company increases ... Zimmer Biomet Holdings, Inc. (NYSE and SIX: ZBH) today ...
(Date:4/28/2016)... , April 28, 2016  ValGenesis, Inc., ... Management Solutions (VLMS) today announced that a ... for sufferers of chronic kidney failure has ... manage their corporate validation process. The global ... software solution to manage their validation processes ...
(Date:4/27/2016)... April 27, 2016 ... Forums in Zürich gab Strekin AG den ... STR001 zur Erhaltung des Resthörvermögens von Patienten, ... Für die umfassende Phase-II-Doppelblindstudie mit Placebo-Kontrollgruppe werden ... STR001 wird während der Operation direkt ins ...
Breaking Medicine Technology:
(Date:5/2/2016)... , ... May 02, 2016 , ... Pregnancy Awareness Month ... trying to conceive. , “If you are ready to have a baby, ... “My book, HealthChequesTM: Journal Babies is your Personal Conception & Pregnancy Organizer, written ...
(Date:5/2/2016)... (PRWEB) , ... May 02, 2016 , ... ... division of Permobil, recently completed two groundbreaking studies that determined that the adjustability ... – are critical in the prevention of pressure injuries, which are now referred ...
(Date:5/2/2016)... ... May 02, 2016 , ... Atlantic Information Services, Inc. (AIS) is pleased ... Model Could Transform the Pharma Landscape .” CMS recently proposed a test of alternate ... provider-administered drugs while preserving care provided to beneficiaries. The webinar will review the details ...
(Date:5/2/2016)... ... , ... The Evolve Paddle Board Company- which specializes in the production and ... their partnership with Yoloha Yoga- producers of high quality cork yoga mats. Together they ... seen a dramatic rise in popularity throughout the last few years. The makers of ...
(Date:5/2/2016)... ... May 02, 2016 , ... Summit BHC (Summit), a leading ... Twin Lakes Recovery Center. Located east of Atlanta in Monroe, Georgia, the ... residential facility is set on 34 acres of beautiful, secluded property that will ...
Breaking Medicine News(10 mins):