Deferiprone
Encyclopedia
Deferiprone is an oral
drug
that chelates iron
and is used to treat thalassaemia major.
It has been licensed for use in Europe
and Asia
for many years while awaiting approval in Canada
and the United States
. On October 14, 2011, however, "the U.S. Food and Drug Administration 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... 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...."
Deferiprone is in clinical trials in the United States to treat Contrast-Induced Acute Kidney Injury and to slow progression of Chronic Kidney Disease; the trials are being conducted by the biotech company, Cormedix.
, and the Hospital for Sick Children
and pharmaceutical giant Apotex
, that started in 1996 and delayed approval of the drug in North America.
Dr. Olivieri's data suggested that deferiprone leads to progressive hepatic fibrosis, a disputed finding.
Mouth
The mouth is the first portion of the alimentary canal that receives food andsaliva. The oral mucosa is the mucous membrane epithelium lining the inside of the mouth....
drug
Drug
A drug, broadly speaking, is any substance that, when absorbed into the body of a living organism, alters normal bodily function. There is no single, precise definition, as there are different meanings in drug control law, government regulations, medicine, and colloquial usage.In pharmacology, a...
that chelates iron
Iron
Iron is a chemical element with the symbol Fe and atomic number 26. It is a metal in the first transition series. It is the most common element forming the planet Earth as a whole, forming much of Earth's outer and inner core. It is the fourth most common element in the Earth's crust...
and is used to treat thalassaemia major.
It has been licensed for use in Europe
Europe
Europe is, by convention, one of the world's seven continents. Comprising the westernmost peninsula of Eurasia, Europe is generally 'divided' from Asia to its east by the watershed divides of the Ural and Caucasus Mountains, the Ural River, the Caspian and Black Seas, and the waterways connecting...
and Asia
Asia
Asia is the world's largest and most populous continent, located primarily in the eastern and northern hemispheres. It covers 8.7% of the Earth's total surface area and with approximately 3.879 billion people, it hosts 60% of the world's current human population...
for many years while awaiting approval in Canada
Canada
Canada is a North American country consisting of ten provinces and three territories. Located in the northern part of the continent, it extends from the Atlantic Ocean in the east to the Pacific Ocean in the west, and northward into the Arctic Ocean...
and the United States
United States
The United States of America is a federal constitutional republic comprising fifty states and a federal district...
. On October 14, 2011, however, "the U.S. Food and Drug Administration 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... 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...."
Deferiprone is in clinical trials in the United States to treat Contrast-Induced Acute Kidney Injury and to slow progression of Chronic Kidney Disease; the trials are being conducted by the biotech company, Cormedix.
Controversy
Deferiprone was at the center of a protracted struggle between Nancy Olivieri, a Canadian haematologist and researcherResearcher
A researcher is somebody who performs research, the search for knowledge or in general any systematic investigation to establish facts. Researchers can work in academic, industrial, government, or private institutions.-Examples of research institutions:...
, and the Hospital for Sick Children
Hospital for Sick Children
The Hospital for Sick Children – is a major paediatric centre for the Greater Toronto Area, serving patients up to age 18. Located on University Avenue in Downtown Toronto, SickKids is part of the city’s Discovery District, a critical mass of scientists and entrepreneurs who are focused on...
and pharmaceutical giant Apotex
Apotex
Apotex is a Canadian pharmaceutical corporation. Founded in 1974, the company is the largest producer of generic drugs in Canada, with sales exceeding one billion dollars a year...
, that started in 1996 and delayed approval of the drug in North America.
Dr. Olivieri's data suggested that deferiprone leads to progressive hepatic fibrosis, a disputed finding.