Deferiprone

Deferiprone

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 category	US: D (Evidence of risk)
Legal status	US: ℞-only
Routes of administration	Oral
Pharmacokinetic data
Metabolism	Glucuronidation
Half-life	2 to 3 hours
Excretion	Renal (75 to 90% in 24 hours)
Identifiers
CAS Registry Number	30652-11-0 Y
ATC code	V03AC02
PubChem	CID 2972
ChemSpider	2866 Y
UNII	2BTY8KH53L Y
KEGG	D07416 Y
ChEBI	CHEBI:68554 N
ChEMBL	CHEMBL70927 Y
Chemical data
Formula	C7H9NO2
Molecular mass	139.152 g/mol
SMILES O=C\1C(\O)=C(/N(/C=C/1)C)C
InChI InChI=1S/C7H9NO2/c1-5-7(10)6(9)3-4-8(5)2/h3-4,10H,1-2H3  Y Key:TZXKOCQBRNJULO-UHFFFAOYSA-N  Y
N (what is this?)  (verify)

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

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 deferiprone leads to progressive hepatic fibrosis.^[6][7]

See also

• Deferoxamine
• Deferasirox

References

1. ↑ 1.0 1.1 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.