Clinical data
Trade names Jakafi, Jakavi
AHFS/ Monograph
MedlinePlus a612006
License data
  • AU: C
  • US: C (Risk not ruled out)
Routes of
Oral, topical
ATC code L01XE18 (WHO)
Legal status
Legal status
Pharmacokinetic data
Bioavailability 95%[1]
Protein binding 97%[1]
Metabolism Hepatic (mainly CYP3A4-mediated)[1]
Biological half-life 2.8-3 hours[1]
Excretion Urine (74%), faeces (22%)[1]
Synonyms INCB018424, INC424
CAS Number 941678-49-5 YesY
ChemSpider 25027389 YesY
Chemical and physical data
Formula C17H18N6
Molar mass 306.37 g/mol
3D model (Jmol) Interactive image
 NYesY (what is this?)  (verify)

Ruxolitinib (trade names Jakafi and Jakavi) is a drug for the treatment of intermediate or high-risk myelofibrosis, a type of myeloproliferative disorder that affects the bone marrow,[2][3] and for polycythemia vera (PCV) when there has been an inadequate response to or intolerance of hydroxyurea.[4][5]

Mechanism of action

Ruxolitinib is a Janus kinase inhibitor with selectivity for subtypes JAK1 and JAK2 of this enzyme.[6][7] Ruxolitinib inhibits dysregulated JAK signaling associated with myelofibrosis. JAK1 and JAK2 recruit signal transducers and activators of transcription (STATs) to cytokine receptors leading to modulation of gene expression.

Side effects

Side effects include thrombocytopenia (low blood platelet count), anemia (low red blood cell count) and neutropenia; risk of infection; symptom exacerbation if the medication is interrupted or discontinued; and non-melanoma skin cancer.[4][8]

Immunologic side effects have included herpes zoster (shingles) and case reports of opportunistic infections.[9] Metabolic side effects have included weight gain. Laboratory abnormalities have included alanine transaminase (ALT) abnormalities, aspartate transaminase (AST) abnormalities, and mildly elevated cholesterol levels.[4]


The phase III Controlled Myelofibrosis Study with Oral JAK Inhibitor-I (COMFORT-I) and COMFORT-II trials showed significant benefits by reducing spleen size and relieving debilitating symptoms.[10][11][12][13]

In November 2011, ruxolitinib was approved by the U.S. Food and Drug Administration (FDA) for the treatment of intermediate or high-risk myelofibrosis based on results of the COMFORT-I and COMFORT-II Trials.[14]

In 2014, it was approved in polycythemia vera (PCV) when there has been an inadequate response to or intolerance of hydroxyurea, based on the RESPONSE trial.[15][5]


It is also being investigated for plaque psoriasis,[6] and for alopecia areata.[16]

In Feb 2016, a phase III trial for pancreatic cancer was terminated due to insufficient efficacy.[17]

Eight weeks-treatment with ruxolitinib blunted senescent cell-mediated inhibition of adipogenesis and increased insulin sensitivity in 22-month-old mice.[18]


  1. 1 2 3 4 5 "Jakafi (ruxolitinib) dosing, indications, interactions, adverse effects, and more". Medscape Reference. WebMD. Retrieved 16 February 2014.
  2. Mesa, Ruben A.; Yasothan, Uma; Kirkpatrick, Peter (2012). "Ruxolitinib". Nature Reviews Drug Discovery. 11 (2): 103–4. doi:10.1038/nrd3652. PMID 22293561.
  3. Harrison, C; Mesa, R; Ross, D; Mead, A; Keohane, C; Gotlib, J; Verstovsek, S (2013). "Practical management of patients with myelofibrosis receiving ruxolitinib". Expert Review of Hematology. 6 (5): 511–23. doi:10.1586/17474086.2013.827413. PMID 24083419.
  5. 1 2 Vannucchi AM, Kiladjian JJ, Griesshammer M et al. (2015). "Ruxolitinib versus standard therapy for the treatment of polycythemia vera". N. Engl. J. Med. 372 (5): 426–35. doi:10.1056/NEJMoa1409002. PMC 4358820Freely accessible. PMID 25629741.
  6. 1 2 Mesa RA (2010). "Ruxolitinib, a selective JAK1 and JAK2 inhibitor for the treatment of myeloproliferative neoplasms and psoriasis". IDrugs. 13 (6): 394–403. PMID 20506062.}
  7. Pardanani, A.; Tefferi, A. (2011). "Targeting myeloproliferative neoplasms with JAK inhibitors". Current Opinion in Hematology. 18 (2): 105–10. doi:10.1097/MOH.0b013e3283439964. PMID 21245760.
  8. Hobbs, GS; Rampal RK (2015). "JAK2 Mutations and JAK Inhibitors in the Management of Myeloproliferative Neoplasms". Contemporary Oncology. 7 (1): 22–28.
  9. Wysham, NG; Allada G; Sullivan DR (2013). "An opportunistic infection associated with ruxolitinib, a novel janus kinase 1,2 inhibitor.". Chest. 143 (5): 1478–9. doi:10.1378/chest.12-1604. PMID 23648912.
  10. Harrison, C.; Kiladjian, J. J.; Al-Ali, H. K.; Gisslinger, H.; Waltzman, R.; Stalbovskaya, V.; McQuitty, M.; Hunter, D. S.; Levy, R.; Knoops, L.; Cervantes, F.; Vannucchi, A. M.; Barbui, T.; Barosi, G. (2012). "JAK Inhibition with Ruxolitinib versus Best Available Therapy for Myelofibrosis". New England Journal of Medicine. 366 (9): 787–798. doi:10.1056/NEJMoa1110556. PMID 22375970.
  11. Verstovsek, S.; Mesa, R. A.; Gotlib, J.; Levy, R. S.; Gupta, V.; Dipersio, J. F.; Catalano, J. V.; Deininger, M.; Miller, C.; Silver, R. T.; Talpaz, M.; Winton, E. F.; Harvey Jr, J. H.; Arcasoy, M. O.; Hexner, E.; Lyons, R. M.; Paquette, R.; Raza, A.; Vaddi, K.; Erickson-Viitanen, S.; Koumenis, I. L.; Sun, W.; Sandor, V.; Kantarjian, H. M. (2012). "A Double-Blind, Placebo-Controlled Trial of Ruxolitinib for Myelofibrosis". New England Journal of Medicine. 366 (9): 799–807. doi:10.1056/NEJMoa1110557. PMID 22375971.
  12. Tefferi, A. (2012). "Challenges Facing JAK Inhibitor Therapy for Myeloproliferative Neoplasms". New England Journal of Medicine. 366 (9): 844–846. doi:10.1056/NEJMe1115119. PMID 22375977.
  13. ASCO Annual Meeting 2011: JAK Inhibitor Ruxolitinib Demonstrates Significant Clinical Benefit in Myelofibrosis Archived November 21, 2011, at the Wayback Machine.
  14. "FDA Approves Incyte's Jakafi (ruxolitinib) for Patients with Myelofibrosis" (Press release). Incyte. Retrieved 2012-01-02.
  15. FDA approval letter.
  16. Falto-Aizpurua, L; Choudhary, S; Tosti, A (December 2014). "Emerging treatments in alopecia.". Expert opinion on emerging drugs. 19 (4): 545–56. PMID 25330928.
  17. Incyte bags late-stage development of Jakafi for solid tumors; shares down 10% premarket. Feb 2016
  18. Xu, Ming; Palmer, Allyson K; Ding, Husheng; Weivoda, Megan M; Pirtskhalava, Tamar; White, Thomas A; Sepe, Anna; Johnson, Kurt O; Stout, Michael B; Giorgadze, Nino; Jensen, Michael D; Lebrasseur, Nathan K; Tchkonia, Tamar; Kirkland, James L (2015). "Targeting senescent cells enhances adipogenesis and metabolic function in old age". ELife. 4. doi:10.7554/eLife.12997.
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