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:
Incyte Investigative Site, Ponce, Puerto Rico
Novartis Investigative Site, Manchester, United Kingdom
Novartis Investigational Site, Bucharest, Romania
Memorial Sloan Kettering Monmouth, Middletown, New Jersey, United States
Memorial Sloan Kettering Commack, Commack, New York, United States
Memorial Sloan Kettering Westchester, Harrison, New York, United States
M D Anderson Cancer Center, Houston, Texas, United States
Mayo Clinic in Arizona, Scottsdale, Arizona, United States
Mayo Clinic in Rochester, Rochester, Minnesota, United States
Northwestern Medicine (Data collection and specimen analysis), Chicago, Illinois, United States
Memorial Sloan Kettering Commack, Commack, New York, United States
Memorial Sloan Kettering Cancer Center, New York, New York, United States
The University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, United States
Wake Forest University Health Sciences, Winston-Salem, North Carolina, United States
Novartis Investigative Site, Kocaeli, Turkey
UCLA Healthcare Hematology-Oncology, Santa Monica, California, United States
New York Oncology Hematology Pc., Clifton Park, New York, United States
Tennessee Oncology, Nashville, Tennessee, United States
Baylor College of Medicine, Houston, Texas, United States
University of Texas MD Anderson Cancer Center, Houston, Texas, United States
University of Alabama at Birmingham, Birmingham, Alabama, United States
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