{"entity":{"id":"ruxolitinib","kind":"drug","name":"Ruxolitinib","aka":[],"tldr":"Ruxolitinib was the first JAK inhibitor: it shrinks the spleen and relieves symptoms in myelofibrosis and controls blood counts in polycythaemia vera, without eliminating the disease clone.","summary":"COMFORT-I/II (2011) showed spleen volume and symptom benefit versus placebo/best available therapy with a later-demonstrated survival advantage; RESPONSE (2014) established it in hydroxyurea-resistant PV. Also approved in acute and chronic graft-versus-host disease (REACH2/3). Anaemia, thrombocytopenia, infections and a discontinuation syndrome are the main issues.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ruxolitinib","links":[{"label":"COMFORT-I (NEJM 2012)","url":"https://doi.org/10.1056/NEJMoa1110557"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ruxolitinib"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera","primary-myelofibrosis","all-ph-like"],"sections":[],"technologies":["kinase-inhibitors","allogeneic-hsct"],"targets":["jak2","jak1"],"drugs":[],"companies":["incyte","novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02955940","nct04562389","nct04509700","nct04817007","nct03669965"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Jakafi / Jakavi","modality":"Small-molecule JAK1/JAK2 inhibitor","mechanism":"ATP-competitive inhibition of JAK1 and JAK2, dampening JAK-STAT signalling regardless of JAK2/CALR/MPL driver.","approvals":[{"region":"US","year":2011,"indication":"Intermediate/high-risk myelofibrosis"},{"region":"US","year":2014,"indication":"Polycythaemia vera after hydroxyurea"},{"region":"US","year":2019,"indication":"Steroid-refractory acute GVHD"},{"region":"US","year":2021,"indication":"Chronic GVHD after failure of 1-2 lines"},{"region":"EU","year":2012,"indication":"Myelofibrosis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/ruxolitinib/","neighbours":{"cancer":[{"id":"cmml","kind":"cancer","name":"Chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms","route":"/cancers/cmml/"},{"id":"essential-thrombocythaemia","kind":"cancer","name":"Essential thrombocythaemia (ET)","route":"/cancers/essential-thrombocythaemia/"},{"id":"myeloproliferative-neoplasms","kind":"cancer","name":"Myeloproliferative neoplasms (PV, ET, myelofibrosis)","route":"/cancers/myeloproliferative-neoplasms/"},{"id":"all-ph-like","kind":"cancer","name":"Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL)","route":"/cancers/all-ph-like/"},{"id":"polycythaemia-vera","kind":"cancer","name":"Polycythaemia vera (PV)","route":"/cancers/polycythaemia-vera/"},{"id":"primary-myelofibrosis","kind":"cancer","name":"Primary myelofibrosis","route":"/cancers/primary-myelofibrosis/"},{"id":"t-large-granular-lymphocytic-leukaemia","kind":"cancer","name":"T-cell large granular lymphocytic leukaemia","route":"/cancers/t-large-granular-lymphocytic-leukaemia/"}],"technology":[{"id":"allogeneic-hsct","kind":"technology","name":"Allogeneic stem cell transplantation","route":"/technologies/allogeneic-hsct/"},{"id":"gvhd-chronic-overview","kind":"technology","name":"Chronic graft-versus-host disease","route":"/technologies/gvhd-chronic-overview/"},{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"},{"id":"gvhd-ruxolitinib-steroid-refractory","kind":"technology","name":"When steroids fail: ruxolitinib for steroid-refractory GvHD","route":"/technologies/gvhd-ruxolitinib-steroid-refractory/"}],"target":[{"id":"calr","kind":"target","name":"CALR","route":"/targets/calr/"},{"id":"jak1","kind":"target","name":"JAK1","route":"/targets/jak1/"},{"id":"jak2","kind":"target","name":"JAK2","route":"/targets/jak2/"},{"id":"mpl","kind":"target","name":"MPL (thrombopoietin receptor)","route":"/targets/mpl/"}],"company":[{"id":"incyte","kind":"company","name":"Incyte","route":"/companies/incyte/"},{"id":"novartis","kind":"company","name":"Novartis","route":"/companies/novartis/"}],"trial":[{"id":"nct04817007","kind":"trial","name":"A Study to Assess the Safety and Tolerability of BMS-986158 Alone and in Combination With Either Ruxolitinib or Fedratinib in Participants With Blood Cancer (Myelofibrosis)","route":"/trials/nct04817007/"},{"id":"aall1521","kind":"trial","name":"AALL1521","route":"/trials/aall1521/"},{"id":"nct02955940","kind":"trial","name":"An Open-Label Study to Enable Continued Treatment Access for Subjects Previously Enrolled in Studies of Ruxolitinib","route":"/trials/nct02955940/"},{"id":"comfort-i","kind":"trial","name":"COMFORT-I","route":"/trials/comfort-i/"},{"id":"comfort-ii","kind":"trial","name":"COMFORT-II","route":"/trials/comfort-ii/"},{"id":"nct04543279","kind":"trial","name":"Fostamatinib as a Single Agent or in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis With Severe Thrombocytopenia","route":"/trials/nct04543279/"},{"id":"nct03669965","kind":"trial","name":"KRT-232 Compared to Ruxolitinib in Patients With Phlebotomy-Dependent Polycythemia Vera","route":"/trials/nct03669965/"},{"id":"majic-et","kind":"trial","name":"MAJIC-ET","route":"/trials/majic-et/"},{"id":"majic-pv","kind":"trial","name":"MAJIC-PV","route":"/trials/majic-pv/"},{"id":"manifest-2","kind":"trial","name":"MANIFEST-2","route":"/trials/manifest-2/"},{"id":"response","kind":"trial","name":"RESPONSE","route":"/trials/response/"},{"id":"response-2","kind":"trial","name":"RESPONSE-2","route":"/trials/response-2/"},{"id":"nct07219576","kind":"trial","name":"Retifanlimab and Ruxolitinib In Solid Malignancies","route":"/trials/nct07219576/"},{"id":"nct04509700","kind":"trial","name":"Rollover Study to Provide Continued Treatment for Participants With B-Cell Malignancies Previously Enrolled in Studies of Parsaclisib (INCB050465)","route":"/trials/nct04509700/"},{"id":"nct04562389","kind":"trial","name":"Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis","route":"/trials/nct04562389/"}],"drug":[{"id":"inca033989","kind":"drug","name":"INCA033989","route":"/drugs/inca033989/"},{"id":"pipobroman","kind":"drug","name":"Pipobroman","route":"/drugs/pipobroman/"}],"paper":[{"id":"paper-comfort-1-ruxolitinib-myelofibrosis-nejm-2012","kind":"paper","name":"COMFORT-I: ruxolitinib, the first JAK inhibitor, versus placebo for myelofibrosis","route":"/key-papers/paper-comfort-1-ruxolitinib-myelofibrosis-nejm-2012/"},{"id":"paper-comfort-ii-ruxolitinib-best-available-therapy-harrison-nejm-2012","kind":"paper","name":"COMFORT-II: JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis","route":"/key-papers/paper-comfort-ii-ruxolitinib-best-available-therapy-harrison-nejm-2012/"},{"id":"paper-momentum-momelotinib-lancet-2023","kind":"paper","name":"MOMENTUM: momelotinib versus danazol for myelofibrosis patients with anaemia after a prior JAK inhibitor","route":"/key-papers/paper-momentum-momelotinib-lancet-2023/"}],"term":[{"id":"aquagenic-pruritus","kind":"term","name":"Aquagenic pruritus","route":"/terms/aquagenic-pruritus/"},{"id":"dipss-mipss70","kind":"term","name":"DIPSS, DIPSS-plus and MIPSS70 (myelofibrosis risk scores)","route":"/terms/dipss-mipss70/"},{"id":"gvhd","kind":"term","name":"Graft-versus-host disease (GVHD) and graft-versus-leukaemia","route":"/terms/gvhd/"},{"id":"jak2-v617f","kind":"term","name":"JAK2 V617F","route":"/terms/jak2-v617f/"},{"id":"mpn-driver-mutations","kind":"term","name":"MPN driver mutations (JAK2 V617F, CALR, MPL) and allele burden","route":"/terms/mpn-driver-mutations/"},{"id":"ph-like-all","kind":"term","name":"Ph-like (BCR::ABL1-like) acute lymphoblastic leukaemia","route":"/terms/ph-like-all/"},{"id":"post-pv-myelofibrosis","kind":"term","name":"Post-PV myelofibrosis (spent phase)","route":"/terms/post-pv-myelofibrosis/"}],"institution":[{"id":"mays-cancer-center","kind":"institution","name":"Mays Cancer Center at UT Health San Antonio","route":"/institutions/mays-cancer-center/"}],"pathway":[{"id":"caf-activation-desmoplasia","kind":"pathway","name":"Fibroblast activation, desmoplasia & matrix stiffness","route":"/pathways/caf-activation-desmoplasia/"}],"idea":[{"id":"idea-pv-clone-directed-therapy","kind":"idea","name":"Clearing the JAK2 clone in polycythaemia vera: interferon plus mutant-selective inhibitors as a route to treatment-free remission","route":"/ideas/idea-pv-clone-directed-therapy/"}]}}