Deferiprone

Deferiprone (tradenames include Ferriprox) is an oral drug that chelates iron and is used to treat thalassaemia major.^[1]

It has been licensed for use in Europe and Asia for many years while awaiting approval in Canada and the United States.^[1][2] 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...."^[3]

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.^[4]

Controversy

Deferiprone was at the center of a protracted struggle between Nancy Olivieri, a Canadian haematologist and researcher, and the Hospital for Sick Children and pharmaceutical giant Apotex, that started in 1996 and delayed approval of the drug in North America.^[5] Dr. Olivieri's data suggested that deferiprone leads to progressive hepatic fibrosis, a disputed finding.^[6][7]

See also

• Deferoxamine
• Deferasirox

References

1. ^ ^{a b} Savulescu J (2004). "Thalassaemia major: the murky story of deferiprone". BMJ 328 (7436): 358–9. doi:10.1136/bmj.328.7436.358. PMC 341373. PMID 14962851. http://www.pubmedcentral.nih.gov/articlerender.fcgi?tool=pmcentrez&artid=341373.  Full Text.
2. ^ http://www.docguide.com/dg.nsf/PrintPrint/F8BC7049D350C2AD852568A2006FEAAC
3. ^ FDA NEWS RELEASE: FDA Approves Ferripox (deferiprone) to Treat Patients with Excess Iron in the Body, Oct. 14, 2011 http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm275814.htm
4. ^ http://www.cormedix.com/pipeline_CRMD001.php
5. ^ Viens A, Savulescu J (2004). "Introduction to The Olivieri symposium.". J Med Ethics 30 (1): 1–7. doi:10.1136/jme.2003.006577. PMC 1757126. PMID 14872065. http://jme.bmj.com/cgi/content/full/30/1/1. 
6. ^ Brittenham G, Nathan D, Olivieri N, Porter J, Pippard M, Vichinsky E, Weatherall D (2003). "Deferiprone and hepatic fibrosis". Blood 101 (12): 5089–90; author reply 5090–1. doi:10.1182/blood-2002-10-3173. PMID 12788794.  Full Text.
7. ^ Wanless I, Sweeney G, Dhillon A, Guido M, Piga A, Galanello R, Gamberini M, Schwartz E, Cohen A (2002). "Lack of progressive hepatic fibrosis during long-term therapy with deferiprone in subjects with transfusion-dependent beta-thalassemia". Blood 100 (5): 1566–9. doi:10.1182/blood-2002-01-0306. PMID 12176871.  Full Text.