CLINICAL TRIALS PROFILE FOR TOFACITINIB

505(b)(2) Clinical Trials for tofacitinib

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Indication	NCT04925973 ↗	Tofacitinib for Hospitalized Acute Severe Ulcerative Colitis Management	Recruiting	McGill University	Phase 2	2021-06-01	The TRIUMPH study was designed to build on the existing literature by studying the efficacy of tofacitinib in hospitalized patients with acute severe ulcerative colitis. This trial will provide evidence for a possible new indication for the use of tofacitinib.
New Indication	NCT04925973 ↗	Tofacitinib for Hospitalized Acute Severe Ulcerative Colitis Management	Recruiting	University of Alberta	Phase 2	2021-06-01	The TRIUMPH study was designed to build on the existing literature by studying the efficacy of tofacitinib in hospitalized patients with acute severe ulcerative colitis. This trial will provide evidence for a possible new indication for the use of tofacitinib.
New Indication	NCT04925973 ↗	Tofacitinib for Hospitalized Acute Severe Ulcerative Colitis Management	Recruiting	University of British Columbia	Phase 2	2021-06-01	The TRIUMPH study was designed to build on the existing literature by studying the efficacy of tofacitinib in hospitalized patients with acute severe ulcerative colitis. This trial will provide evidence for a possible new indication for the use of tofacitinib.
New Indication	NCT04925973 ↗	Tofacitinib for Hospitalized Acute Severe Ulcerative Colitis Management	Recruiting	University of Manitoba	Phase 2	2021-06-01	The TRIUMPH study was designed to build on the existing literature by studying the efficacy of tofacitinib in hospitalized patients with acute severe ulcerative colitis. This trial will provide evidence for a possible new indication for the use of tofacitinib.

All Clinical Trials for tofacitinib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00413699 ↗	Long-Term Effectiveness And Safety Of CP-690,550 For The Treatment Of Rheumatoid Arthritis	Completed	Pfizer	Phase 3	2007-02-05	The purpose of this study is to determine the long-term effectiveness and safety of CP-690,550 for the treatment of rheumatoid arthritis. Subjects are eligible for this study only after participating in another "qualifying" study of CP-690,550 A sub-study will be conducted within the A3921024 study, this study will evaluate the immune response to pneumococcal and influenza vaccines in patients receiving CP-690,550
NCT01164579 ↗	Effects of Tofacitinib (CP-690,550) on Magnetic Resonance Imaging (MRI)- Assessed Joint Structure In Early Rheumatoid Arthritis (RA)	Completed	Pfizer	Phase 2	2010-10-01	Evaluation of efficacy and safety of tofacitinib (CP-690,550) for the treatment of early rheumatoid arthritis in adult patients with moderate to severe disease who are methotrexate naïve. The efficacy will be evaluated by exploring the effects on joint structure assessed by magnetic resonance imaging, x-rays and by standard clinical assessment.
NCT01375127 ↗	Collection of Follow-up Data From CP-690,550-treated Kidney Transplant Recipients	Completed	Pfizer		2011-08-01	This is an observational study designed to collect follow-up clinical date on subjects who were treated with tofacitinib in 2 completed Phase 2 studies who either discontinued treatment prematurely or did not elect to enroll in long-term extension studies.
NCT01458951 ↗	A Study To Evaluate Both The Efficacy and Safety Profile of CP-690,550 In Patients With Moderately to Severely Active Ulcerative Colitis	Completed	Pfizer	Phase 3	2012-06-01	This study is designed to evaluate the efficacy and safety of tofacitinib (CP-690,550) in patients with moderate to severe ulcerative colitis who have failed or be intolerant to one of following treatments for ulcerative colitis: oral steroids, azathiopurine/6-mercaptopurine, or anti-TNF-alpha therapy.

Clinical Trial Conditions for tofacitinib

Condition Name

Condition Name for tofacitinib
Intervention	Trials
Rheumatoid Arthritis	30
Healthy	11
Ulcerative Colitis	9
Psoriatic Arthritis	8

Condition MeSH

Condition MeSH for tofacitinib
Intervention	Trials
Arthritis	49
Arthritis, Rheumatoid	39
Colitis, Ulcerative	12
Arthritis, Psoriatic	11

Clinical Trial Locations for tofacitinib

Trials by Country

Trials by Country for tofacitinib
Location	Trials
United States	461
China	94
Mexico	69
Canada	59
Australia	51

Trials by US State

Trials by US State for tofacitinib
Location	Trials
California	29
Florida	28
Texas	25
Connecticut	20
Pennsylvania	20

Clinical Trial Progress for tofacitinib

Clinical Trial Phase

Clinical Trial Phase for tofacitinib
Clinical Trial Phase	Trials
PHASE4	10
PHASE3	5
PHASE2	6

Clinical Trial Status

Clinical Trial Status for tofacitinib
Clinical Trial Phase	Trials
Completed	59
Recruiting	48
Not yet recruiting	23

Clinical Trial Sponsors for tofacitinib

Sponsor Name

Sponsor Name for tofacitinib
Sponsor	Trials
Pfizer	50
Shanghai Zhongshan Hospital	6
Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh	5

Sponsor Type

Sponsor Type for tofacitinib
Sponsor	Trials
Other	140
Industry	84
NIH	6

Tofacitinib: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Tofacitinib, marketed under the brand name Xeljanz, is a Janus kinase (JAK) inhibitor developed by Pfizer. It primarily treats autoimmune disorders, notably rheumatoid arthritis (RA), psoriatic arthritis, and ulcerative colitis. As a pioneering oral targeted therapy, tofacitinib's market trajectory depends on ongoing clinical trials, evolving regulatory landscapes, and competitive innovations within immunology and inflammatory diseases.

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Clinical Trials Update

Current Status and Indications

Since its approval in 2012 by the U.S. Food and Drug Administration (FDA), tofacitinib has undergone multiple clinical investigations to expand therapeutic indications and assess long-term safety. The pivotal Phase III trials, such as ORAL Solo and ORAL Step, established its efficacy in RA patients refractory to conventional DMARDs[1]. Notably, these trials demonstrated significant improvements in American College of Rheumatology (ACR) response criteria, radiographic progression, and patient-reported outcomes.

Recent and Ongoing Trials

In recent years, Pfizer has focused on expanding tofacitinib’s indications and confirming its safety profile through extensive Phase III and Phase IV programs:

• Ulcerative Colitis: The OCTAVE trials (OCTAVE Induction 1 & 2, OCTAVE Sustain) have demonstrated tofacitinib’s efficacy in inducing and maintaining remission in moderate-to-severe ulcerative colitis. Data show remission rates significantly higher than placebo, with notable improvements in mucosal healing[2][3].

• Pediatric Rheumatology: Pfizer has initiated studies like Pincinn to evaluate safety and efficacy in adolescent populations with juvenile idiopathic arthritis (JIA). Preliminary results indicate promising activity warranting further investigation.

• Other Autoimmune Conditions: Trials are exploring tofacitinib’s potential in alopecia areata, dermatologic conditions, and inflammatory bowel disease (IBD). A notable Phase II trial is evaluating tofacitinib in Crohn’s disease.

Safety and Regulatory Developments

Regulatory agencies have issued warnings over safety concerns, particularly risks of serious infections, thromboembolism, and gastrointestinal perforations. The FDA released a boxed warning and limited indications in 2019, emphasizing cautious use. Concomitantly, post-marketing surveillance continues to monitor long-term safety, influencing ongoing trial designs and labeling updates.

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Market Analysis

Market Landscape and Competitive Position

Tofacitinib has established itself as a key player in the oral immunomodulator segment, competing primarily with biologics like adalimumab, etanercept, and newer oral agents—such as upadacitinib (AbbVie) and filgotinib (Gilead). The convenience of oral administration gives tofacitinib a competitive edge over injectable biologics, attracting patient and physician preference—particularly in RA and ulcerative colitis.

Current Market Size

The global tofacitinib market was valued at approximately $1.2 billion in 2022, with expectations of compound annual growth rate (CAGR) around 8% through 2030[4]. Factors influencing growth include:

• Increasing prevalence of autoimmune diseases, notably RA, affecting over 1.3 million Americans alone.
• Growing demand for oral targeted therapies over traditional biologics.
• Broadened indications, especially in ulcerative colitis and other immune-modulating conditions.

Geographic and Demographic Insights

The U.S. dominates the market due to high adoption, regulatory approval, and extensive clinical data. Europe exhibits rapid growth driven by expanding healthcare infrastructure and supportive regulatory pathways. Emerging markets, particularly in Asia-Pacific, show increasing adoption, supported by expanding healthcare access and rising disease prevalence.

Challenges and Limitations

Regulatory safety warnings impact market growth—restrictions and new contraindications could hinder uptake. Healthcare providers exhibit cautious prescribing, especially in patients at risk for infections or cardiovascular events.

Market Drivers

• Patient preference for oral therapies.
• Expansion into new autoimmune indications validated by clinical trials.
• Strategic collaborations and approvals—such as the FDA’s approval of tofacitinib for ulcerative colitis in 2018—boosting confidence in its safety profile.

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Future Market Projections

Growth Drivers

The market is poised for significant expansion over the next decade, driven by:

• Broader indications, with ongoing trials in IBD, PsA, JIA, and alopecia areata.
• Incremental improvement in safety profiles, likely through formulation advances or combination therapies.
• Increased awareness and diagnosis of autoimmune conditions amid aging populations.

Potential Risks

• Safety concerns remain central. Post-marketing adverse effects could lead to regulatory restrictions.
• Competitive threat from newer, more selective JAK inhibitors like upadacitinib (Rinvoq) and filgotinib (Jyseleca) that are gaining market share due to better safety profiles or efficacy.
• Patents risk expiry could lead to generic competition, pressuring prices.

Forecast Overview

By 2030, global tofacitinib sales are projected to reach approximately $3.5 billion, with CAGR estimates around 9%. The continued expansion of approved indications, combined with strategic product positioning, will be pivotal for Pfizer’s market share.

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Regulatory Outlook and Strategic Positioning

Pfizer continues to prioritize the expansion of tofacitinib's label, including potential approval for psoriasis and Crohn’s disease, pending successful trial outcomes. Collaborative efforts with regulatory agencies aim to optimize benefit-risk assessments and streamline approval pathways. Additionally, real-world evidence plays a crucial role in post-approval monitoring and market expansion strategies.

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Key Takeaways

• Diverse Clinical Portfolio: Tofacitinib remains central to Pfizer's portfolio, with ongoing trials in ulcerative colitis, juvenile idiopathic arthritis, and emerging indications like alopecia areata.
• Market Growth: The global market is on a robust growth trajectory, fueled by the demand for oral immunomodulators, despite safety concerns and competition.
• Safety and Regulatory Challenges: Vigilance over safety signals influences clinical development and marketing strategies. Clear communication with healthcare providers about risks is vital.
• Competitive Landscape: Advancements from competitors like upadacitinib threaten to erode market share; Pfizer must innovate in safety profiles and indications.
• Future Potential: Continuous clinical innovation and strategic regulatory submissions are essential for maintaining relevance and competitive advantage through 2030.

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FAQs

Q1: What are the primary indications for tofacitinib currently approved by regulators?
A: Tofacitinib is approved for moderate-to-severe rheumatoid arthritis, active psoriatic arthritis, and ulcerative colitis in various regions, including the U.S. and EU.

Q2: What safety concerns have influenced tofacitinib’s market and regulatory status?
A: Serious infections, thromboembolism, gastrointestinal perforations, and elevated risk of malignancies have led to boxed warnings and restricted indications, impacting prescribing patterns.

Q3: What are the key competitive advantages of tofacitinib?
A: The oral route of administration, proven efficacy in multiple autoimmune conditions, and expanding indication approvals position it favorably.

Q4: How do ongoing clinical trials impact the future of tofacitinib?
A: Trials exploring new indications, pediatric use, and safety enhancements could significantly broaden its market footprint and stimulate growth.

Q5: What is the projected market size for tofacitinib in 2030?
A: Estimated at approximately $3.5 billion globally, driven by new indications and increased global adoption.

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References

[1] Lee DW, et al. "Clinical Efficacy of Tofacitinib in Rheumatoid Arthritis." Arthritis & Rheumatology, 2016.
[2] Sandborn WJ, et al. "A Randomized Trial of Tofacitinib for Ulcerative Colitis." N Engl J Med, 2017.
[3] Panés J, et al. "Safety and Efficacy Data from OCTAVE Trials." Gastroenterology, 2020.
[4] MarketLine. "Global Immunology Drugs Market Report," 2022.

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