Ruxolitinib, formerly known as INCB018424 or INC424, is an anticancer drug and a Janus kinase (JAK) inhibitor. It is a potent and selective inhibitor of JAK1 and JAK2, which are tyrosine kinases involved in cytokine signalling and hematopoiesis. Myeloproliferative neoplasms, such as myelofibrosis and polycythemia vera, are often characterized by aberrant activation of the JAK-STAT pathway, leading to abnormal blood cell counts and thrombotic complications. By inhibiting JAK1 and JAK2, ruxolitinib works to block the dysregulated cell signalling pathways and prevents abnormal blood cell proliferation. Due to a large number of patients with myeloproliferative neoplasms who have JAK2 mutations, ruxolitinib was the first ATP-competitive inhibitor of JAK1 and JAK2 ever developed.
Ruxolitinib was first approved for the treatment of adult patients with myelofibrosis by the FDA in 2011, followed by EMA's approval in 2012. In 2014, it was approved for the treatment of polycythemia vera in adults who have an inadequate response to or are intolerant of hydroxyurea and in 2019, ruxolitinib was approved for use in steroid-refractory acute graft-versus-host disease in adults and children. The topical formulation of ruxolitinib is used to treat atopic dermatitis and vitiligo. It is being investigated for other inflammatory skin conditions.
Ruxolitinib has been investigated to treat patients with coronavirus disease 2019 (COVID-19) accompanied by severe systemic hyperinflammation. In phase II clinical trials, ruxolitinib improved chest computed tomography and improved recovery in patients with lymphopenia. However, phase III clinical trials later determined that ruxolitinib was inadequate in meeting its primary endpoint of reducing the number of hospitalized COVID-19 patients who experienced severe complications thus the drug was not approved as a treatment for COVID-19.
Ruxolitinib is indicated for the treatment of the following conditions:
Topical ruxolitinib is indicated for:
West China Hospital Sichuan University, Chengdu, Sichuan, China
Hematology Hospital, Chinese Academy of Medical Sciences, Tianjin, Tianjin, China
Policlinico Sant'Orsola-Malpighi, Bologna, Italy
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia, Brescia, Italy
Azienda Ospedaliero Universitaria Policlinico "G. Rodolico - San Marco", Catania, Italy
Froedtert & the Medical College of Wisconsin, Milwaukee, Wisconsin, United States
Mayo Clinic Hospital, Phoenix, Arizona, United States
City of Hope Medical Center, Duarte, California, United States
MD Anderson Cancer Center, Houston, Texas, United States
MD Anderson Cancer Center, Houston, Texas, United States
University of Chicago Medicine Comprehensive Cancer Center, Chicago, Illinois, United States
Duke, Durham, North Carolina, United States
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