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Clinical data
Trade namesJakafi, Jakavi
SynonymsINCB018424, INC424
License data
  • AU: C
  • US: C (Risk not ruled out)
Routes of
By mouth, topical
ATC code
Legal status
Legal status
Pharmacokinetic data
Protein binding97%[1]
MetabolismHepatic (mainly CYP3A4-mediated)[1]
Elimination half-life2.8-3 hours[1]
ExcretionUrine (74%), faeces (22%)[1]
CAS Number
PubChem CID
PDB ligand
CompTox Dashboard (EPA)
Chemical and physical data
Molar mass306.37 g/mol g·mol−1
3D model (JSmol)
 ☒N☑Y (what is this?)  (verify)

Ruxolitinib (trade names Jakafi /ˈækəf/ JAK-ə-fye 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] Ruxolitinib has also been shown to improve cases of chronic graft versus host disease in patients following a bone marrow transplant. It was developed and is currently sold by Incyte Corp.

Mechanism of action[edit]

Ruxolitinib is a janus kinase inhibitor (JAK inhibitor) with selectivity for subtypes JAK1 and JAK2.[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[edit]

Side effects include pancytopenia,[citation needed] 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] alopecia areata,[16] relapsed diffuse large B-cell lymphoma, and peripheral T-cell lymphoma.[17]

In Feb 2016, a phase III trial for pancreatic cancer was terminated due to insufficient efficacy.[18]

Eight weeks-treatment with ruxolitinib blunted senescent cell-mediated inhibition of adipogenesis and increased insulin sensitivity in 22-month-old mice.[19]

Topical ruxolitinib as a cream applied to vitiligo-affected skin sometimes is effective at repigmenting the skin[20]

As of September 2019. a clinical trial was in progress to evaluate "Treatment Free Remission After Combination Therapy With Ruxolitinib Plus Tyrosine Kinase Inhibitors".[21]


  1. ^ a b c d e "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. ^ a b 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 4358820. PMID 25629741.CS1 maint: uses authors parameter (link)
  6. ^ a b 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.CS1 maint: uses authors parameter (link)
  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. PMC 5991580. 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. PMC 4822164. 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. doi:10.1517/14728214.2014.974550. PMID 25330928.
  17. ^ Vose, Julie. "Ruxolitinib Phosphate (Oral JAK Inhibitor INCB18424) in Treating Patients With Relapsed or Refractory Diffuse Large B-Cell or Peripheral T-Cell Non-Hodgkin Lymphoma".
  18. ^ Incyte bags late-stage development of Jakafi for solid tumors; shares down 10% premarket. Feb 2016
  19. ^ 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: e12997. doi:10.7554/eLife.12997. PMC 4758946. PMID 26687007.
  20. ^ Existing drug for blood problems is used to treat vitiligo
  21. ^