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Ruxolitinib

Generic Name
Ruxolitinib
Brand Names
Jakafi, Jakavi, Opzelura
Drug Type
Small Molecule
Chemical Formula
C17H18N6
CAS Number
941678-49-5
Unique Ingredient Identifier
82S8X8XX8H
Background

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.

Indication

Ruxolitinib is indicated for the treatment of the following conditions:

Topical ruxolitinib is indicated for:

Associated Conditions
Acute Graft-Versus-Host Disease (GVHD), Chronic Graft-Versus-Host Disease, Non-segmental Vitiligo, Post Polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis (Post-ET MF), Primary Myelofibrosis (PMF), High risk Myelofibrosis, Intermediate risk Myelofibrosis, Mild Atopic dermatitis, Moderate Atopic dermatitis, Refractory Polycythemia vera
Associated Therapies
-

Flunotinib Maleate Phase II Intermediate-High Risk Bone Fiber Clinical Trial

Phase 2
Recruiting
Conditions
MF,PMF,PPV-MF,PET-MF
Interventions
Drug: Flonoltinib 50mg
Drug: Flonoltinib 100mg
Drug: Ruxolitinib
First Posted Date
2024-06-13
Last Posted Date
2024-06-13
Lead Sponsor
Chengdu Zenitar Biomedical Technology Co., Ltd
Target Recruit Count
75
Registration Number
NCT06457425
Locations
🇨🇳

West China Hospital Sichuan University, Chengdu, Sichuan, China

🇨🇳

Hematology Hospital, Chinese Academy of Medical Sciences, Tianjin, Tianjin, China

RVU120 in Patients with Intermediate or High-risk, Primary or Secondary Myelofibrosis

Phase 2
Recruiting
Conditions
Myelofibrosis
Interventions
Drug: RVU120
Drug: Ruxolitinib
First Posted Date
2024-05-02
Last Posted Date
2024-12-17
Lead Sponsor
Ryvu Therapeutics SA
Target Recruit Count
230
Registration Number
NCT06397313
Locations
🇮🇹

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

and more 15 locations

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

Phase 2
Recruiting
Conditions
Chronic Graft-versus-host-disease
Interventions
First Posted Date
2024-04-29
Last Posted Date
2024-12-30
Lead Sponsor
Incyte Corporation
Target Recruit Count
120
Registration Number
NCT06388564
Locations
🇺🇸

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

and more 67 locations

Efficacy and Safety of Anti-CD25 rhMAb in the Treatment of Steroid-Refractory cGVHD

Phase 4
Not yet recruiting
Conditions
cGVHD
Interventions
First Posted Date
2024-04-15
Last Posted Date
2024-04-15
Lead Sponsor
Peking University People's Hospital
Target Recruit Count
118
Registration Number
NCT06364319

High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly

Phase 2
Recruiting
Conditions
Splenomegaly
Myelofibrosis
Interventions
First Posted Date
2024-04-03
Last Posted Date
2024-11-05
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
30
Registration Number
NCT06345495
Locations
🇺🇸

MD Anderson Cancer Center, Houston, Texas, United States

Open Label Phase 2 Study of Tasquinimod in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)

Phase 2
Recruiting
Conditions
Primary Myelofibrosis
Post-polycythemia Vera Myelofibrosis
Post-Essential Thrombocytosis Myelofibrosis
Interventions
First Posted Date
2024-03-25
Last Posted Date
2024-08-05
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
33
Registration Number
NCT06327100
Locations
🇺🇸

MD Anderson Cancer Center, Houston, Texas, United States

Ivosidenib and Ruxolitinib in Patients With Advanced Myeloproliferative Neoplasms (MPNs) That Have an IDH1 Gene Mutation

Phase 1
Recruiting
Conditions
Myeloproliferative Neoplasms
Interventions
First Posted Date
2024-03-04
Last Posted Date
2024-10-16
Lead Sponsor
University of Chicago
Target Recruit Count
18
Registration Number
NCT06291987
Locations
🇺🇸

University of Chicago Medicine Comprehensive Cancer Center, Chicago, Illinois, United States

JAK Inhibitor in Acquired Hemophagocytic synDrome in the Intensive Care Unit

Phase 2
Not yet recruiting
Conditions
Hemophagocytic Syndromes
Interventions
First Posted Date
2024-02-06
Last Posted Date
2024-02-06
Lead Sponsor
Assistance Publique - Hôpitaux de Paris
Target Recruit Count
42
Registration Number
NCT06244862

Ruxolitinib Plus Fostamatinib for Steroid Refractory cGvHD

Phase 1
Not yet recruiting
Conditions
Chronic Graft Versus Host Disease
Interventions
First Posted Date
2024-01-31
Last Posted Date
2024-06-11
Lead Sponsor
Stefanie Sarantopoulos, MD, PhD.
Target Recruit Count
30
Registration Number
NCT06233110
Locations
🇺🇸

Duke, Durham, North Carolina, United States

A Trial of Selinexor, Ruxolitinib and Methylprednisolone

Phase 1
Not yet recruiting
Conditions
Multiple Myeloma in Relapse
Multiple Myeloma, Refractory
Interventions
First Posted Date
2024-01-25
Last Posted Date
2024-01-25
Lead Sponsor
Oncotherapeutics
Target Recruit Count
30
Registration Number
NCT06225310
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