Deferiprone

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Deferiprone
Deferiprone.svg
Systematic (IUPAC) name
3-hydroxy-1,2-dimethylpyridin-4(1H)-one
Clinical data
Trade names Ferriprox
AHFS/Drugs.com International Drug Names
Licence data EMA:Link, US FDA:link
Pregnancy cat. D (US)
Legal status -only (US)
Routes Oral
Pharmacokinetic data
Metabolism Glucuronidation
Half-life 2 to 3 hours
Excretion Renal (75 to 90% in 24 hours)
Identifiers
CAS number 30652-11-0 YesY
ATC code V03AC02
PubChem CID 2972
ChemSpider 2866 YesY
UNII 2BTY8KH53L YesY
KEGG D07416 YesY
ChEBI CHEBI:68554 N
ChEMBL CHEMBL70927 YesY
Chemical data
Formula C7H9NO2 
Mol. mass 139.152 g/mol
 N (what is this?)  (verify)

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

In 1994 was first approved for use in treating thalassaemia major in 1994.[2] and had been licensed for use in Europe and Asia for many years while awaiting approval in Canada and the United States.[1][3] On October 14, 2011, it was approved for use in the US under the FDA’s accelerated approval program.[4]

Controversy[edit]

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.[6][7]

See also[edit]

References[edit]

  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.  Full Text.
  2. ^ Staff, Cipla. Cipla's History
  3. ^ http://www.docguide.com/dg.nsf/PrintPrint/F8BC7049D350C2AD852568A2006FEAAC
  4. ^ 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
  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. 
  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.