Deferiprone

Deferiprone
Deferiprone
Systematic (IUPAC) name
3-hydroxy-1,2-dimethylpyridin-4(1H)-one
Clinical data
AHFS/Drugs.com International Drug Names
Licence data EMA:LinkUS 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
ChEMBL CHEMBL70927 YesY
Chemical data
Formula C7H9NO2 
Mol. mass 139.152 g/mol
SMILES eMolecules & PubChem
 YesY(what is this?)  (verify)

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

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.

Wikimedia Foundation. 2010.

Игры ⚽ Поможем написать курсовую

Look at other dictionaries:

  • deferiprone — pl.m. deferiproni …   Dizionario dei sinonimi e contrari

  • DFP — • deferiprone; • diastolic filling period; • diisopropyl fluorophosphate …   Dictionary of medical acronyms & abbreviations

  • Nancy Fern Olivieri — Nancy Fern Olivieri, BSc, MD, FRCPC, is a prominent Toronto haematologist and researcher with an interest in the treatment of hemoglobinopathies. She is best known for a protracted struggle with the Hospital for Sick Children and the… …   Wikipedia

  • Mittelmeeranämie — Klassifikation nach ICD 10 D56.0 Alpha Thalassämie D56.1 Beta Thalassämie D56.2 Delta Beta Thalassämie …   Deutsch Wikipedia

  • Mittelmeerkrankheit — Klassifikation nach ICD 10 D56.0 Alpha Thalassämie D56.1 Beta Thalassämie D56.2 Delta Beta Thalassämie …   Deutsch Wikipedia

  • Thalassämia major — Klassifikation nach ICD 10 D56.0 Alpha Thalassämie D56.1 Beta Thalassämie D56.2 Delta Beta Thalassämie …   Deutsch Wikipedia

  • Thalassämia minor — Klassifikation nach ICD 10 D56.0 Alpha Thalassämie D56.1 Beta Thalassämie D56.2 Delta Beta Thalassämie …   Deutsch Wikipedia

  • Thalassämie — Klassifikation nach ICD 10 D56.0 Alpha Thalassämie D56.1 Beta Thalassämie D56.2 Delta Be …   Deutsch Wikipedia

  • Thalassémie — Les thalassémies, encore appelées dans leur forme majeure anémie ou maladie de Cooley, sont des formes d anémies héréditaires associées à une hémoglobinopathie (déficience dans la synthèse d une ou de plusieurs des quatre chaînes formant l… …   Wikipédia en Français

  • Deferasirox — Systematic (IUPAC) name [4 [(3Z,5E) 3,5 bis(6 oxo 1 cyclohexa 2,4 dienylidene) 1,2,4 triazolidin 1 yl]benzoic acid Clinical data Licence …   Wikipedia

Share the article and excerpts

Direct link
Do a right-click on the link above
and select “Copy Link”