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Systematic (IUPAC) name
3'-{(2Z)-2-[1-(3,4-dimethylphenyl)-3-methyl-5-oxo-1,5-dihydro-4H-pyrazol-4-ylidene]hydrazino}-2'-hydroxy-3-biphenylcarboxylic acid
Clinical data
AHFS/Drugs.com monograph
MedlinePlus a609011
Licence data US FDA:link
  • AU: B3
  • US: C
496775-61-2 N
496775-62-3 (olamine)
PubChem CID 9846180
ChemSpider 21106301 YesY
Chemical data
Formula C25H22N4O4
442.467 g/mol
 N (what is this?)  (verify)

Eltrombopag (rINN, codenamed SB-497115-GR) is a medication that has been developed for conditions that lead to thrombocytopenia (abnormally low platelet counts). It is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin. Eltrombopag was discovered as a result of research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals. Designated an orphan drug in the USA and European Union, it is being manufactured and marketed by GlaxoSmithKline under the trade name Promacta in the USA and will be marketed as Revolade in the EU. Eltrombopag was approved by the U.S. Food and Drug Administration on November 20, 2008.[1] Eltrombopag received FDA breakthrough treatment designation in February 2014 for patients with aplastic anemia for which immunosuppression has not been successful.[2] It has been shown to produce a trilineage hematopoesis in some patients with aplastic anemia, resulting in increased platelet counts, along with red and white blood cell counts.[3]


In preclinical studies, the compound was shown to interact selectively with the thrombopoeitin receptor, leading to activation of the JAK-STAT signaling pathway and increased proliferation and differentiation of megakaryocytes. Animal studies confirmed that administration could increase platelet counts. In 73 healthy volunteers, higher doses of eltrombopag caused larger increases in the number of circulating platelets without tolerability problems.[4]

Clinical trials[edit]

Eltrombopag has been studied, and shown to be effective, in two major clinical syndromes: idiopathic thrombocytopenic purpura[5] and cirrhosis due to hepatitis C (in which low platelet counts may be a contraindication for interferon treatment).[6] After 6 weeks of therapy in a phase III trial, eltrombopag 50 mg/day was associated with a significantly higher response rate than placebo in adult patients with chronic ITP.[7]


  1. ^ "FDA approves Promacta (eltrombopag), the first oral medication to increase platelet production for people with serious blood disorder" (Press release). GlaxoSmithKline. 2008-11-20. Retrieved 2008-11-25. 
  2. ^ "GSK gains FDA Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for severe aplastic anaemia". GSK Press Release. GSK. Retrieved 28 March 2014. 
  3. ^ Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M et al. (Mar 2014). "Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug". Blood 123 (12): 1818–25. doi:10.1182/blood-2013-10-534743. PMID 24345753. 
  4. ^ Jenkins JM, Williams D, Deng Y, Uhl J, Kitchen V, Collins D et al. (Jun 2007). "Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist". Blood 109 (11): 4739–41. doi:10.1182/blood-2006-11-057968. PMID 17327409. 
  5. ^ Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B et al. (Nov 2007). "Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura". The New England Journal of Medicine 357 (22): 2237–2247. doi:10.1056/NEJMoa073275. PMID 18046028. 
  6. ^ McHutchison JG, Dusheiko G, Shiffman ML, Rodriguez-Torres M, Sigal S, Bourliere M et al. (Nov 2007). "Eltrombopag for thrombocytopenia in patients with cirrhosis associated with hepatitis C". The New England Journal of Medicine 357 (22): 2227–2236. doi:10.1056/NEJMoa073255. PMID 18046027. 
  7. ^ Garnock-Jones KP, Keam SJ (2009). "Eltrombopag". Drugs 69 (5): 567–76. doi:10.2165/00003495-200969050-00005. PMID 19368418.