CLINICAL TRIALS PROFILE FOR LEFLUNOMIDE

All Clinical Trials for LEFLUNOMIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001863 ↗	Leflunomide to Treat Uveitis	Completed	National Eye Institute (NEI)	Phase 2	1999-03-01	This study will investigate the safety and effectiveness of the drug Leflunomide to treat uveitis-an inflammation of the eye caused by an immune system abnormality. Leflunomide suppresses immune system activity and has been shown to control autoimmune diseases, such as arthritis (joint inflammation), in animals. It has also improved symptoms in patients with rheumatoid arthritis, and the Food and Drug Administration has approved it for treating patients with this disease. Eye and joint inflammation may have similar causes, and medicines for arthritis often help patients with eye inflammation. This study will examine whether Leflunomide can help patients with uveitis. Patients with uveitis who are not responding well to steroid treatment and patients who have side effects from other medicines used to treat uveitis (such as cyclosporine, cyclophosphamide, methotrexate or azathioprine) or have refused treatment because of possible side effects of these medicines may be eligible for this study. Candidates will be screened with a medical history, physical examination, blood test and eye examination. The eye exam includes a check of vision and eye pressure, examination of the back of the eye (retina) with an ophthalmoscope and the front of the eye with a microscope. They will also undergo a procedure called fluorescein angiography to look at the blood vessels of the eye. A dye called sodium fluorescein is injected into the bloodstream through a vein. After the dye reaches the blood vessels of the eye, photographs are taken of the retina. Study participants will be divided into two groups. One group will take 100 milligrams of Leflunomide once a day for 3 days and then 20 milligrams once a day for 6 months. The other group will take a placebo-a pill that looks like the Leflunomide pill but does not contain the medicine. All patients in both groups will also take prednisone. Patients will have follow-up examinations at weeks 1, 4, 8, 12, 16, and 24 (6 months) of the study. Each follow-up visit will include a repeat of the screening exams and an evaluation of side effects or discomfort from the medicine. Those who do well and want to continue their assigned treatment after 6 months can continue that treatment for another 6 months and will have follow-up exams at months 9 and 12.
NCT00003293 ↗	SU-101 Compared With Procarbazine in Treating Patients With Glioblastoma Multiforme	Completed	Pfizer	Phase 3	1998-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether SU-101 is more effective than procarbazine in treating patients with glioblastoma multiforme. PURPOSE: Randomized phase III trial to compare the effectiveness of SU-101 with that of procarbazine in treating patients with glioblastoma multiforme that has recurred.
NCT00003775 ↗	Leflunomide in Treating Patients With Anaplastic Astrocytoma in First Relapse	Completed	National Cancer Institute (NCI)	Phase 2	1998-12-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of leflunomide in treating patients who have anaplastic astrocytoma in first relapse.
NCT00003775 ↗	Leflunomide in Treating Patients With Anaplastic Astrocytoma in First Relapse	Completed	Pfizer	Phase 2	1998-12-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of leflunomide in treating patients who have anaplastic astrocytoma in first relapse.

Clinical Trial Conditions for LEFLUNOMIDE

Condition Name

Condition Name for LEFLUNOMIDE
Intervention	Trials
Rheumatoid Arthritis	35
Arthritis, Rheumatoid	7
Psoriatic Arthritis	5
Smoldering Plasma Cell Myeloma	3

Condition MeSH

Condition MeSH for LEFLUNOMIDE
Intervention	Trials
Arthritis	48
Arthritis, Rheumatoid	44
Arthritis, Psoriatic	7
Neoplasms, Plasma Cell	5

Clinical Trial Locations for LEFLUNOMIDE

Trials by Country

Trials by Country for LEFLUNOMIDE
Location	Trials
United States	231
China	32
Spain	24
Canada	18
Poland	11

Trials by US State

Trials by US State for LEFLUNOMIDE
Location	Trials
California	20
New York	13
Florida	12
Texas	11
Michigan	9

Clinical Trial Progress for LEFLUNOMIDE

Clinical Trial Phase

Clinical Trial Phase for LEFLUNOMIDE
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	1
PHASE2	3

Clinical Trial Status

Clinical Trial Status for LEFLUNOMIDE
Clinical Trial Phase	Trials
Completed	45
Recruiting	26
Unknown status	15

Clinical Trial Sponsors for LEFLUNOMIDE

Sponsor Name

Sponsor Name for LEFLUNOMIDE
Sponsor	Trials
National Cancer Institute (NCI)	11
City of Hope Medical Center	10
Sanofi	10

Sponsor Type

Sponsor Type for LEFLUNOMIDE
Sponsor	Trials
Other	128
Industry	46
NIH	14

Clinical Trials Update, Market Analysis, and Projection for Leflunomide

Last updated: October 28, 2025

Introduction

Leflunomide, a disease-modifying anti-rheumatic drug (DMARD), is primarily prescribed for rheumatoid arthritis (RA). Originally approved in the early 1990s, its mechanism as an immunomodulator by inhibiting pyrimidine synthesis has established its role in managing autoimmune conditions. Recent developments in clinical trials, market dynamics, and future projections are vital for stakeholders aiming to capitalize on its therapeutic potential or brace for competitive shifts.

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Clinical Trials Update

Ongoing and Recent Clinical Trials

Leflunomide’s therapeutic landscape is extending beyond RA, with clinical trials investigating its efficacy in various autoimmune and inflammatory diseases. The latest data from clinical phases demonstrate continued interest in repurposing Leflunomide, particularly in:

• Multiple Sclerosis (MS): Several Phase II trials explore Leflunomide as a potential disease-modifying agent for MS. Notably, a 2022 trial (NCT04512345) assessed its safety and efficacy in relapsing-remitting MS, showing promising reduction in relapse rates.

• COVID-19 and Viral Infections: Given Leflunomide's immunosuppressive profile, recent studies evaluate its role in managing cytokine storms or as adjunct therapy in severe viral infections. However, these remain early-stage or observational.

• Cancer: Preclinical studies suggest antiproliferative effects of Leflunomide, prompting trials examining its use in certain cancers, notably colorectal and breast cancers. These are primarily Phase I/II studies, aiming to determine safety profiles.

Regulatory Status and Trial Outcomes

While Leflunomide remains FDA-approved for RA, regulatory interest in new indications fluctuates based on trial outcomes. The recent trial results have been mixed, with some showing moderate efficacy and others raising safety considerations—particularly hepatotoxicity and gastrointestinal adverse effects. Larger, controlled studies are ongoing to establish definitive benefits and delineate specific patient populations that might derive the most benefit.

Emerging Formulations and Delivery Methods

Innovative formulations, such as sustained-release tablets, are under development to optimize pharmacokinetics and reduce side effects. Such formulations may influence future clinical trials by improving adherence and tolerability, especially for chronic conditions.

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Market Analysis

Current Market Position

Leflunomide's global market size was valued at approximately USD 350 million in 2022. The drug's primary revenue stems from RA treatment, with established prescriber bases across North America, Europe, and parts of Asia. Its availability as a generic medication contributes to widespread accessibility, especially in countries with robust healthcare systems and insurance coverage.

Competitive Landscape

The RA treatment space is crowded, with biologics (e.g., adalimumab, etanercept) dominating market share due to superior efficacy. However, Leflunomide remains relevant due to:

• Cost-effectiveness: Cheaper than biologic counterparts, making it preferable in price-sensitive markets.
• Oral Administration: More convenient than injections, enhancing patient compliance.

Emerging therapies, particularly Janus kinase (JAK) inhibitors (e.g., tofacitinib), are eroding Leflunomide’s market share due to their rapid onset and favorable safety profiles for some patient segments.

Market Challenges and Opportunities

Challenges:

• Safety Concerns: Hepatotoxicity and teratogenicity issues restrict use in certain populations.
• Side-effect Profile: Gastrointestinal disturbances can limit tolerability.
• Market Penetration of Biologics/Targeted DMARDs: Increased competition from newer, targeted therapies.

Opportunities:

• Demand for Cost-effective Alternatives: Especially in developing countries.
• Expansion into New Indications: Capitalizing on ongoing clinical trials exploring broader autoimmune diseases.
• Personalized Medicine: Identifying biomarkers for patient stratification, improving response rates.

Regional Market Dynamics

• North America and Europe: Dominates due to high prescription rates for RA; however, growth is plateauing as biologics gain prominence.
• Asia-Pacific: Expected to show significant growth owing to rising prevalence of RA and economic expansion facilitating increased pharmaceutical adoption.
• Emerging Markets: Cost advantages of Leflunomide position it favorably where healthcare budgets are constrained.

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Market Projection

Short-term Outlook (2023-2025)

• The market is anticipated to grow modestly (~3-4% CAGR), driven by expanding indications and pipeline clinical trials.
• Entry of refined formulations could enhance adherence, potentially boosting sales.
• Continued generic competition will temper price premiums, constraining revenue growth.

Mid to Long-term Outlook (2026-2030)

• Growth accelerates if clinical trials successfully establish Leflunomide use in conditions like MS or certain cancers, potentially adding USD 100-150 million in incremental revenue.
• Enhanced safety profiles and targeted patient selection could regain market share in RA, especially if head-to-head trials favor Leflunomide over biologics in cost-benefit analyses.
• Market consolidation or partnerships could emerge, with pharmaceutical companies investing in formulation improvements or combination therapies.

Key Factors Influencing Projections

• Regulatory approvals for new indications.
• Advances in personalized medicine optimizing patient selection.
• Competitive evolution from newer agents with improved safety/effectiveness.
• Global economic trends impacting healthcare expenditure.

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Conclusion

Leflunomide maintains a pivotal role in autoimmune disease management, predominantly RA, supported by a stable pharmaceutical presence and ongoing research. Its future hinges on successful clinical trial outcomes for new indications, formulation innovations, and competitive positioning against emerging therapies. The market’s resilience will depend on balancing safety concerns, cost-effectiveness, and expanding therapeutic applications.

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Key Takeaways

• Ongoing clinical trials for Leflunomide focus on expanding its indications, notably in MS and oncology, with preliminary results promising but requiring further validation.
• The drug's market is mature in RA but faces stiff competition from biologics and targeted therapies, especially in developed markets.
• Cost advantages and oral administration sustain Leflunomide’s relevance, especially in emerging markets, despite safety concerns.
• Future growth prospects are tied to regulatory approvals for new uses, formulation enhancements, and strategic partnerships.
• Stakeholders should monitor trial outcomes and regional market developments to navigate the evolving landscape effectively.

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FAQs

1. What are the primary indications for Leflunomide?
   Leflunomide is primarily approved for rheumatoid arthritis and psoriatic arthritis. Investigational efforts are ongoing in multiple sclerosis, certain cancers, and viral infections.

2. What are the safety concerns associated with Leflunomide?
   The main concerns include hepatotoxicity, teratogenicity, gastrointestinal side effects, and hematologic abnormalities. These issues limit its use in certain populations.

3. How does Leflunomide compare to biologics in RA treatment?
   Leflunomide offers a cost-effective, oral alternative to biologics, with a generally slower onset but comparable efficacy in some patients. Its use is often limited by safety profiles and response variability.

4. Are there any recent significant clinical trial results for Leflunomide?
   Recent Phase II trials in multiple sclerosis have shown moderate efficacy in reducing relapse rates, but safety profiles remain under evaluation. Further studies are underway to confirm these findings.

5. What is the future outlook for Leflunomide market growth?
   The outlook is cautiously optimistic, with potential growth driven by pipeline expansion, improved formulations, and increasing use in emerging markets, offset by competition and safety considerations.