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.
New Indication	NCT04925973 ↗	Tofacitinib for Hospitalized Acute Severe Ulcerative Colitis Management	Recruiting	McMaster 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.

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.
NCT01465763 ↗	A Study Evaluating The Efficacy And Safety Of CP-690,550 In Patients With Moderate To Severe Ulcerative Colitis	Completed	Pfizer	Phase 3	2012-04-01	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	29
Healthy	11
Ulcerative Colitis	8
Psoriatic Arthritis	8

Condition MeSH

Condition MeSH for Tofacitinib
Intervention	Trials
Arthritis	48
Arthritis, Rheumatoid	34
Colitis	11
Ulcer	10

Clinical Trial Locations for Tofacitinib

Trials by Country

Trials by Country for Tofacitinib
Location	Trials
United States	451
China	86
Mexico	69
Canada	58
Spain	51

Trials by US State

Trials by US State for Tofacitinib
Location	Trials
California	28
Florida	27
Texas	23
Connecticut	20
Pennsylvania	20

Clinical Trial Progress for Tofacitinib

Clinical Trial Phase

Clinical Trial Phase for Tofacitinib
Clinical Trial Phase	Trials
Phase 4	26
Phase 3	28
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Tofacitinib
Clinical Trial Phase	Trials
Completed	55
Recruiting	39
Not yet recruiting	23

Clinical Trial Sponsors for Tofacitinib

Sponsor Name

Sponsor Name for Tofacitinib
Sponsor	Trials
Pfizer	50
Yale University	4
Shanghai Zhongshan Hospital	4

Sponsor Type

Sponsor Type for Tofacitinib
Sponsor	Trials
Other	109
Industry	79
NIH	6

Tofacitinib: Clinical Trials, Market Analysis, and Projections

Introduction to Tofacitinib

Tofacitinib, marketed under the brand name XELJANZ, is a Janus kinase (JAK) inhibitor used to treat various autoimmune and inflammatory conditions, including rheumatoid arthritis, ulcerative colitis, and psoriasis. Here, we will delve into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials of Tofacitinib

Efficacy and Safety Studies

The efficacy and safety of tofacitinib have been extensively tested in several clinical trials. For instance, six clinical trials involving adult patients with moderate to severe rheumatoid arthritis (RA) were conducted. These trials included 1,589 participants treated with XELJANZ 5 mg twice daily. The studies assessed the drug's ability to reduce symptoms of RA, such as the number of tender and swollen joints, and lab test results indicating inflammation[4].

Key Findings and Safety Concerns

A notable clinical trial comparing tofacitinib with tumour necrosis factor (TNF) inhibitors found an increased risk of serious side effects associated with tofacitinib. These side effects included major cardiovascular problems, cancer, blood clots in the lungs and deep veins, serious infections, and death. This trial involved patients aged 50 and older with at least one cardiovascular risk factor[1].

Regulatory Implications

The findings from these clinical trials have led to new safety warnings. For example, the Therapeutic Goods Administration (TGA) issued a safety alert highlighting the increased risk of serious side effects with tofacitinib, particularly in older patients with cardiovascular risk factors[1].

Market Analysis of Tofacitinib

Current Market Size and Growth

The tofacitinib market has experienced rapid growth in recent years. In 2023, the market size was valued at $2.76 billion and is projected to grow to $3.12 billion in 2024, with a compound annual growth rate (CAGR) of 13.0%. By 2028, the market is expected to reach $5.02 billion, driven by expanded indications, market access initiatives, and real-world evidence[2].

Market Segmentation

The tofacitinib market is segmented by drug class, strength, route of administration, distribution channel, and application. The drug classes include antirheumatic, JAK inhibitors, and immunosuppressants. The strengths available are 5mg, 10mg, 11mg, and 22mg, with oral and other routes of administration. Distribution channels include hospital pharmacies, retail pharmacies, and online pharmacies. The primary applications are ulcerative colitis, rheumatoid arthritis, and psoriasis[2][3].

Regional and Country Analysis

The global tofacitinib market is split by region and country, with significant growth anticipated in various regions due to the increasing prevalence of autoimmune diseases. The Asia-Pacific region, in particular, is expected to contribute significantly to the market growth[2].

Market Drivers and Restraints

Drivers

• Increasing Prevalence of Autoimmune Diseases: The rise in autoimmune illnesses such as rheumatoid arthritis, psoriatic arthritis, and ulcerative colitis drives the demand for tofacitinib. For example, over 52.5 million adult Americans have been diagnosed with arthritis or another rheumatic disease, and this number is expected to increase to 67 million by 2030[2].
• Rising Geriatric Population: Autoimmune diseases are more common in the elderly, and the growing geriatric population is expected to increase the demand for tofacitinib[3].
• Increasing R&D Activities: Ongoing research and development in the areas of autoimmune disorders and JAK inhibitors are driving market growth[3].

Restraints

• Strict Regulations: Drug approval is subject to strict regulatory standards, which can delay market entry and limit market growth[3].
• Adverse Effects and Safety Concerns: The increased risk of serious side effects associated with tofacitinib, as identified in clinical trials, poses a significant restraint to the market[1].

Future Projections and Trends

Market Growth Projections

The tofacitinib market is expected to continue its rapid growth. By 2030, the market is projected to reach $5.67 billion, with a CAGR of 4.7% during the forecast period of 2023 to 2030[3].

Emerging Trends

• Focus on Pediatric Rheumatoid Arthritis Treatment: There is an increasing focus on treating pediatric rheumatoid arthritis, which is expected to drive market growth[2].
• Exploration in Dermatological Conditions: Tofacitinib is being explored for its potential in treating dermatological conditions, which could expand its market[2].
• Strategic Collaborations and Partnerships: Pharmaceutical companies are engaging in strategic collaborations and partnerships to enhance the development and delivery of tofacitinib[2].
• Integration of Real-World Evidence: Real-world evidence is being integrated into treatment guidelines, which is expected to support the growth of the tofacitinib market[2].

Personalized Medicine and Technological Advancements

Growing Demand for Personalized Medicine

Personalized medicine strategies, which involve creating treatment plans based on genetic differences and biomarkers, present opportunities for enhancing the effectiveness of tofacitinib. This approach can be explored through partnerships with diagnostic businesses[3].

Rising Technological Advancements

Improvements in drug development, such as enhanced formulations and novel methodologies, are expected to increase the effectiveness, patient compliance, and administration convenience of tofacitinib[3].

Key Takeaways

• Tofacitinib has shown efficacy in treating autoimmune and inflammatory conditions but is associated with serious side effects, particularly in older patients.
• The market for tofacitinib is growing rapidly, driven by the increasing prevalence of autoimmune diseases and ongoing R&D activities.
• The market is segmented by drug class, strength, route of administration, distribution channel, and application.
• Future growth is expected to be driven by expanded indications, strategic collaborations, and the integration of real-world evidence.
• Personalized medicine and technological advancements are key trends that will shape the future of the tofacitinib market.

FAQs

What are the primary applications of tofacitinib?

Tofacitinib is primarily used to treat ulcerative colitis, rheumatoid arthritis, and psoriasis.

What are the serious side effects associated with tofacitinib?

Tofacitinib is associated with serious side effects including major cardiovascular problems, cancer, blood clots in the lungs and deep veins, serious infections, and death, particularly in older patients with cardiovascular risk factors[1].

What is the projected market size of tofacitinib by 2030?

The tofacitinib market is projected to reach $5.67 billion by 2030, with a CAGR of 4.7% during the forecast period of 2023 to 2030[3].

What are the main drivers of the tofacitinib market?

The main drivers include the increasing prevalence of autoimmune diseases, the rising geriatric population, and ongoing R&D activities in the areas of autoimmune disorders and JAK inhibitors[3].

How is personalized medicine expected to impact the tofacitinib market?

Personalized medicine strategies are expected to enhance the effectiveness of tofacitinib by creating treatment plans based on genetic differences and biomarkers, which can be explored through partnerships with diagnostic businesses[3].

Sources

1. Therapeutic Goods Administration. "New safety warning for medicines used in arthritis and other inflammatory conditions." Retrieved May 11, 2023.
2. The Business Research Company. "Tofacitinib Market Report 2024."
3. Data Bridge Market Research. "Global Tofacitinib Market – Industry Trends and Forecast to 2030."
4. XELJANZ. "Clinical Trials | XELJANZ® (tofacitinib) For RA | Safety Info."