Drug ID:Drug112
Drug Name:Tofacitinib
CID:9926791
DrugBank ID:DB08895
Modality:Small Molecule
Groups:approved
US Approved:YES
Other Approved:YES
Identifier: NCT00787202, , NCT03281304, , NCT01458574, , NCT01470612, , NCT06625450, , NCT04424303, , NCT05112263, , NCT06095128, , NCT05728008, , NCT05431283, , NCT05313620, , NCT04925973, , NCT06780683, , NCT06274996, , NCT05782907, , NCT06095596, , NCT03653026, , NCT02819635, , NCT03345836, , NCT03006
Molecular Formula:C16H20N6O
Molecular Weight:312.37 g/mol
Isomeric SMILES:C[C@@H]1CCN(C[C@@H]1N(C)C2=NC=NC3=C2C=CN3)C(=O)CC#N
Synonyms:Tofacitinib; 477600-75-2; 3-((3R,4R)-4-methyl-3-(methyl(7H-pyrrolo[2,3-d]pyrimidin-4-yl)amino)piperidin-1-yl)-3-oxopropanenitrile; Cp-690 free base; tofacitinibum; UNII-87LA6FU830; CHEBI:71200; 87LA6FU830; CP-690550 FREE BASE; CP-690,550 FREE BASE
Phase 0: 6
Phase 1: 84
Phase 2: 131
Phase 3: 80
Phase 4: 39
Description:Tofacitinib is an inhibitor of Janus kinases, a group of intracellular enzymes involved in signaling pathways that affect hematopoiesis and immune cell function. It is approved by the FDA for treatment of moderate to severe rheumatoid arthritis that responds inadequately to methotrexate or in those who are intolerant to methotrexate. Besides rheumatoid arthritis, tofacitinib has also been studied in clinical trials for the prevention of organ transplant rejection, and the treatment of psoriasis and ulcerative colitis. Known adverse effects include nausea and headache as well as more serious immunologic and hematological adverse effects. Tofacitinib is marketed under the brand name Xeljanz.

Molecular Structure

2D Molecular Structure

Knowledge Graph

dtIDCIDCompound NameGene IDGene NameSpeciesPubMed IDsAction
dt948 9926791 Tofacitinib 2048 EPHB2 Homo sapiens (human) Inhibitor
dt949 9926791 Tofacitinib 6777 STAT5B Homo sapiens (human) None
dt950 9926791 Tofacitinib 3716 JAK1 Homo sapiens (human) Inhibitor
dt951 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) None
dt952 9926791 Tofacitinib 29110 TBK1 Homo sapiens (human) 25540906 None
dt953 9926791 Tofacitinib 3717 JAK2 Homo sapiens (human) 24417533 Inhibition
dt954 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) Inhibition
dt955 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) 24417533 Inhibition
dt956 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) 27572962 Inhibition
dt957 9926791 Tofacitinib 3716 JAK1 Homo sapiens (human) 24417533 Inhibition

  • No data available

Phase Distribution
Phase Description
  • Phase 0: Exploratory trials to assess drug behavior in humans
  • Phase 1: Safety trials to determine safe dosage range
  • Phase 2: Efficacy trials to evaluate therapeutic effects
  • Phase 3: Large-scale trials to confirm efficacy and safety
  • Phase 4: Post-marketing surveillance for long-term safety and efficacy

Trial IDTitlePhaseStatusSponsorIndicationsInterventions
NCT04624230 Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis PHASE3 RECRUITING Pfizer Ulcerative Colitis DRUG: tofacitinib Details
NCT01465763 A Study Evaluating The Efficacy And Safety Of CP-690,550 In Patients With Moderate To Severe Ulcerative Colitis PHASE3 COMPLETED Pfizer Ulcerative Colitis DRUG: tofacitinib|DRUG: Placebo Details
NCT04032756 Tofacitinib Registry of Patients With Ulcerative Colitis in Germany None TERMINATED Ced Service GmbH Colitis, Ulcerative|Biologics|Tofacitiniib|Chroni… None Details
NCT01458951 A Study To Evaluate Both The Efficacy and Safety Profile of CP-690,550 In Patients With Moderately to Severely Active Ulcerative Colitis PHASE3 COMPLETED Pfizer Ulcerative Colitis DRUG: tofacitinib|DRUG: Placebo Details
NCT05112263 Tofacitinib Versus Cyclosporine for Steroid Refractory Acute Severe Ulcerative Colitis None NOT_YET_RECRUITING Post Graduate Institute of Medical Education and Research, Chandigarh Ulcerative Colitis DRUG: Tofacitinib|DRUG: Cyclosporine Details
NCT06469424 An Active Surveillance, Post-Authorization Study to Characterize the Safety of Tofacitinib in Patients With Moderately to Severely Active Ulcerative Colitis in the Real-World Setting Using Data From the United Registries for Clinical Assessment and Research (UR-CARE) in the European Union (EU) None RECRUITING Grupo Espanol de Trabajo en Enfermedad de Crohn y Colitis Ulcerosa Ulcerative Colitis None Details
NCT06095128 A Study of Vedolizumab With Tofacitinib in Adults With Ulcerative Colitis (UC) PHASE4 RECRUITING Takeda Ulcerative Colitis DRUG: Vedolizumab|DRUG: Tofacitinib Details
NCT05728008 Retrospective Observational Comparison Study Between Ustekinumab and Tofacitinib as Third Line Therapy in a Multicenter Cohort of Patients With Refractory Ulcerative Colitis. None COMPLETED IRCCS San Raffaele Ulcerative Colitis DRUG: Ustekinumab|DRUG: Tofacitinib Details
NCT04743518 Impact of Anti-TNF, Vedolizumab and Tofacitinib on Aortic Stiffness, Carotid Intima-media Thickness and Cardiovascular Risk of Patients With Ulcerative Colitis None UNKNOWN Centre Hospitalier Universitaire, Amiens Ulcerative Colitis OTHER: Aortic pulse wave velocity|OTHER: carotid … Details
NCT05104723 Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications PHASE1|PHASE2 ENROLLING_BY_INVITATION National Institute of Allergy and Infectious Diseases (NIAID) Chronic Granulomatous Disease|Inflammatory Gastro… DRUG: XELJANZ (tofacitinib) Details
NCT05431283 Real-world Effectiveness of Tofacitinib on Ulcerative Colitis Associated Spondyloarthropathy None UNKNOWN Italian Group for the study of Inflammatory Bowel Disease (IG-IBD) Ulcerative Colitis|Spondyloarthropathy DRUG: Tofacitinib Details
NCT03103412 TD-3504 SAD in Healthy Subjects and Subjects With Ulcerative Colitis (UC) PHASE1 COMPLETED Theravance Biopharma Active Mild Ulcerative Colitis, Active Moderate U… DRUG: TD-3504|DRUG: 15N2-tofacitinib|DRUG: Placebo Details
NCT05069259 Generate Real World Data On Tofacitinib Induction Therapy and Changes In Clinical and Patient Reported Outcomes. None TERMINATED Pfizer Ulcerative Colitis OTHER: Stool sample collection Details
NCT03663400 Identify Predictors That Distinguish Between Tofacitinib Responders and Non-responders Based on Genotype and Cellular and Molecular Profiles From Pinch Biopsies, Blood and Stool Samples None COMPLETED NYU Langone Health Ulcerative Colitis DIAGNOSTIC_TEST: GWAS analysis by Illumina BeadCh… Details
NCT05313620 Effect of Tofacitinib on Coagulation and Platelet Function, and Its Role in Thromboembolic Events PHASE4 RECRUITING Fundación de Investigación Biomédica - Hospital Universitario de La Princesa Ulcerative Colitis|Thromboembolism DRUG: Tofacitinib|DRUG: Infliximab Adalimumab y G… Details
CTRI/2024/01/061649 Prevalence and impact of sarcopenia and frailty in inflammatory bowel disease, and the effect of multi-modal interventions on sarcopenia and clinical outcomes in patients with inflammatory bowel disease - NIL PHASE3 Not Recruiting All India Institute of Medical Sciences Health Condition 1: K509- Crohns disease, unspeci… Intervention1: High protein anti-inflammatory die… Details
CTRI/2023/10/058514 Evaluation of effectiveness and safety of oral tofacitinib in patients with Parthenium dermatitis: A prospective interventional open label study - NIL Not Available Not Recruiting AIIMS, New Delhi Health Condition 1: L237- Allergic contact dermat… Intervention1: Oral Tofacitinib: Tofacitinib is a… Details
ISRCTN96296121 Defining microbial predictors of responsiveness to biologic therapies in Crohn disease and ulcerative colitis Not Available Recruiting Newcastle upon Tyne Hospitals NHS Foundation Trust Crohn disease and ulcerative colitis Digestive S… Current interventions as of 19/12/2024: This is… Details
ISRCTN13495664 SARS-CoV2 Vaccination immunogenicity in Immunosuppressed inflammatory bowel disease Patients Not Available Not Recruiting Imperial College London Immune responses to SARS-CoV-2 vaccination in imm… 600 IBD patients will be recruited stratified acc… Details
CTRI/2021/10/037641 An open label study to compare the efficacy and safety of tofacitinib compared to corticosteroids for inducing remission in moderately severe active ulcerative colitis PHASE4 Recruiting Dayanand Medical College and Hospital Ludhiana Disease.2. Total mayo clinic score <6 or >9. 3.… Health Condition 1: K51- Ulcerative colitis Details

Disease IDDisease NameDefinitionCategoryRelated DrugsMechanism
No data available

Strategy IDTherapeutic StrategySynonymsRelated DrugsMechanism
S07 Blockade of cytokine Cytokine signalling apremilast; roflumilast; filgotinib; tofacitinib; ABT494; ABT-874; ustekinumab; isankizumab; LY-2525623; AMG139; MEDI2079; guselkumab Various T-cell subsets, their differentiation pathways and … Details

Tofacitinib

PMID: 21171673
Year: 2010
Relationship Type: Treatment Score: 7.1

Tofacitinib (CP-690,550; CP-690550; CP690550), an orally active immunosuppressant, is being developed by Pfizer for the treatment of rheumatoid arthr…

Prospective observational study of tofacitinib in ulcerative colitis - analysis…

PMID: 40535533
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib is a Janus kinase inhibitor for the treatment of ulcerative colitis (UC). Prospective real-world data are scarce. OBJECTIVES:…

Real-life effectiveness and safety of tofacitinib and vedolizumab as 2nd-line f…

PMID: 40335332
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND AND AIMS: Drug positioning in ulcerative colitis (UC) patients refractory to anti-tumor necrosis factor (TNF) is still debated. In a natio…

Differential effects of tofacitinib on macrophage activation contribute to lack…

PMID: 40320713
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND AND AIMS: Tofacitinib, a Janus kinase inhibitor, is approved for the treatment of moderate-to-severe ulcerative colitis. Nonetheless, 40-6…

Post-marketing surveillance of tofacitinib in patients with ulcerative colitis …

PMID: 40259041
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND: We present the final analysis of a tofacitinib post-marketing surveillance (PMS) study in Japanese patients with ulcerative colitis (UC).…

Drug tissue concentration and STAT3 modulation as determinants of tofacitinib r…

PMID: 40243193
Year: 2025
Relationship Type: Treatment Score: 6.5

INTRODUCTION: Inflammatory bowel disease management has advanced with therapies like Janus kinase inhibitors (JAKi). Despite their promise, JAKi phar…

Postoperative Outcomes in Tofacitinib-Treated Patients With Acute Severe Ulcera…

PMID: 40239733
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND AND AIMS: Up to 30% of patients with acute severe ulcerative colitis (ASUC) will require urgent colectomy despite initiation of intravenou…

Inflammatory bowel disease therapeutics: a bibliometric analysis of tofacitinib…

PMID: 40235538
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND: Ulcerative colitis (UC) is a chronic inflammatory bowel disease affecting millions worldwide. Tofacitinib, an inhibitor of Janus kinase (…

Restoration of colonic barrier function by tofacitinib in experimental colitis:…

PMID: 40159830
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND/AIMS: Inflammatory bowel disease can be triggered by disturbances in intestinal mucosal integrity, leading to bacterial transmigration. Th…

Real-World Experience With Tofacitinib in Steroid-Dependent Patients With Moder…

PMID: 40130137
Year: 2025
Relationship Type: Treatment Score: 6.5

INTRODUCTION: Patients with moderate-to-severe ulcerative colitis (UC) who are steroid-dependent despite being on immunomodulators pose significant c…

Effect of tofacitinib exposure in utero and during breastfeeding on the immune …

PMID: 40127036
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND AND AIMS: Janus kinase (JAK) inhibitors, such as tofacitinib, are potent anti-inflammatory agents used in the treatment of ulcerative coli…

Tofacitinib for ulcerative colitis in Brazil: a multicenter observational study…

PMID: 40102788
Year: 2025
Relationship Type: Treatment Score: 6.5

AIM: To assess the real-life, long-term effectiveness and safety of tofacitinib in a large cohort of patients with refractory or difficult-to-treat u…

Upadacitinib Results in Endoscopic Remission in Patients With Inflammatory Bowe…

PMID: 40063408
Year: 2025
Relationship Type: Treatment Score: 6.5

GOALS: Assess the safety and effectiveness of upadacitinib in patients with prior tofacitinib failure. BACKGROUND: Patients with severe, refractory C…

Potential predictors of efficacy outcomes following tofacitinib dose reduction …

PMID: 40028509
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib is an oral Janus kinase inhibitor for the treatment of ulcerative colitis (UC). OBJECTIVES: To investigate potential predicto…

Efficacy and safety of tofacitinib for the treatment of moderate-to-severe ulce…

PMID: 39976073
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND AND AIMS: Tofacitinib has been approved for the treatment of patients with moderate-to-severe ulcerative colitis independently of prior th…

Activated type 17 helper T cells affect tofacitinib treatment outcomes

PMID: 39971758
Year: 2025
Relationship Type: Treatment Score: 6.5

The incidence of ulcerative colitis (UC) is on the rise also in Japan. Simultaneously, therapeutic options, including biologics and Janus kinase (JAK…

Tofacitinib for the treatment of chronic pouchitis: A pilot study

PMID: 39895228
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND & AIMS: Up to 15% of ulcerative colitis (UC) patients with an ileo-anal pouch will develop chronic or antibiotic refractory pouchitis. We …

Tofacitinib and budesonide treatment affect stemness and chemokine release in I…

PMID: 39885201
Year: 2025
Relationship Type: Treatment Score: 6.5

Restoration of the intestinal epithelial barrier is crucial for achieving mucosal healing, the therapeutic goal for inflammatory bowel disease (IBD).…

Continuous Treatment with Tofacitinib but Not Filgotinib Is Effective in Non-Re…

PMID: 39797304
Year: 2025
Relationship Type: Treatment Score: 6.5

Background: Few studies have compared the efficacy and safety of Janus kinase (JAK) inhibitors in patients with ulcerative colitis (UC). We compared …

Tofacitinib Mitigates the Increased SARS-CoV-2 Infection Susceptibility Caused …

PMID: 39756517
Year: 2025
Relationship Type: Treatment Score: 6.5

BACKGROUND & AIMS: Coronavirus disease (COVID-19), caused by severe acquired respiratory syndrome-Coronavirus-2 (SARS-CoV-2), triggered a global pand…

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