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Ball-and-stick model of the halofuginone molecule
Clinical data
AHFS/Drugs.comInternational Drug Names
ATCvet code
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Legal status
  • Investigational
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Chemical and physical data
Molar mass414.68 g·mol−1
3D model (JSmol)
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Halofuginone is a coccidiostat used in veterinary medicine. It is a synthetic halogenated derivative of febrifugine, a natural quinazolinone alkaloid which can be found in the Chinese herb Dichroa febrifuga (Chang Shan).[1] Collgard Biopharmaceuticals is developing halofuginone for the treatment of scleroderma and it has received orphan drug designation from the U.S. Food and Drug Administration.[2]

Halofuginone inhibits the development of T helper 17 cells, immune cells that play an important role in autoimmune disease, but it does not affect other kinds of T cells which are involved in normal immune function.[3] Halofuginone therefore has potential for the treatment of autoimmune disorders.[4]

Halofuginone is also an inhibitor of collagen type I gene expression and as a consequence it may inhibit tumor cell growth.[1] Halofuginone exerts its effects by acting as a high affinity inhibitor of the enzyme glutamyl-prolyl tRNA synthetase. Inhibition of prolyl tRNA charging leads to the accumulation of uncharged prolyl tRNAs, which serve as a signal to initiate the amino acid starvation response, which in turn exerts anti-inflammatory and anti-fibrotic effects.[5]


  1. ^ a b Halofuginone hydrobromide, NCI Drug Dictionary
  2. ^ Halofuginone Receives FDA Orphan Drug Status For Scleroderma Archived 2012-09-12 at WebCite, March 10, 2000
  3. ^ Sundrud, M. S.; Koralov, S. B.; Feuerer, M.; Calado, D. P.; Kozhaya, A. E.; Rhule-Smith, A.; Lefebvre, R. E.; Unutmaz, D.; Mazitschek, R. (2009). "Halofuginone Inhibits TH17 Cell Differentiation by Activating the Amino Acid Starvation Response". Science. 324 (5932): 1334–8. Bibcode:2009Sci...324.1334S. doi:10.1126/science.1172638. PMC 2803727. PMID 19498172.
  4. ^ A new lead for autoimmune disease: A small-molecule drug inhibits Th17 cells, eases symptoms in mouse model, June 4, 2009
  5. ^ Keller, Tracy L; Zocco, Davide; Sundrud, Mark S; Hendrick, Margaret; Edenius, Maja; Yum, Jinah; Kim, Yeon-Jin; Lee, Hak-Kyo; Cortese, Joseph F; Wirth, Dyann F; Dignam, John David; Rao, Anjana; Yeo, Chang-Yeol; Mazitschek, Ralph; Whitman, Malcolm (2012). "Halofuginone and other febrifugine derivatives inhibit prolyl-tRNA synthetase". Nature Chemical Biology. 8 (3): 311. doi:10.1038/nchembio.790. PMC 3281520. PMID 22327401.