CLINICAL TRIALS PROFILE FOR AXITINIB

505(b)(2) Clinical Trials for AXITINIB

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 Combination	NCT03571438 ↗	Evaluation of a Promising New Combination of Protein Kinase Inhibitors on Organotypic Cultures of Human Renal Tumors	Recruiting	University Hospital, Grenoble	N/A	2017-10-16	The investigators objective is to test the combination directly on organotypic cultures of tumors from patients after their excision in the Department of Urology and Renal Transplantation of the University Hospital of Grenoble and to compare their efficacy with that of currently selected treatments in the clinic. The population targeted by the combination for use in clinical practice is patients with metastatic clear cell renal cell carcinoma. Current treatments for these patients are Sunitinib, Pazopanib and Temsirolimus.
New Combination	NCT05070221 ↗	Study of Oncolytic Virus in Combination With HX-008 and Axitinib in Melanoma Patients With Liver Metastasis	Not yet recruiting	Beijing Cancer Hospital	Phase 1	2021-10-01	Malignant melanoma, is a kind of malignant tumor derived from melanocytes. It is common in skin, mucous membrane, eye choroid and other parts. Melanoma is one of the fastest growing malignant tumors with an annual incidence rate of 3-5%. In 2012, there were 232000 new cases of melanoma and 55000 deaths worldwide. Though, the incidence rate of melanoma is relatively low in China, it has been increasing rapidly in recent years. Melanoma has seriously endangering the health of Chinese people. Patients with stage Ⅳ melanoma have a poor prognosis. According to statistics, the median survival time of stage M1a melanoma is 15 months, while stage M1b is 8 months. The median survival time of bone metastasis melanoma is 6 months, while liver and brain metastasis is 4 months. The overall median survival time of metastatic melanoma is only 7.5 months, and the 2-year survival rate is 15%. For patients with advanced melanoma, dacarbazine is the only chemotherapy drug approved by NMPA, but its overall effective rate is only 13.4%, and the median survival time is 5.6 ~ 11 months. Therapies(new drugs or new combination treatments)with higher remission rate and longer survival are urgently needed for patients with advanced melanoma.
New Combination	NCT07159191 ↗	Envafolimab Combined With Axitinib as First-Line Treatment for Patients With Advanced Renal Cell Carcinoma	NOT_YET_RECRUITING	Peking Union Medical College Hospital	PHASE2	2025-09-01	Introduction: This document explains a clinical research study conducted at Peking Union Medical College Hospital. The study aims to evaluate a new combination treatment - Envafolimab (an immunotherapy) and Axitinib (a targeted therapy) - for patients newly diagnosed with advanced kidney cancer (specifically, clear cell Renal Cell Carcinoma or RCC). This information is designed to help patients, their families, and healthcare providers understand the study's purpose, procedures, potential benefits and risks, and what participation involves. 1. What is the Study About? * The Problem: Advanced kidney cancer (RCC) that has spread (metastatic) or cannot be removed by surgery (unresectable) is challenging to treat. While treatments exist, researchers are always looking for more effective and manageable options, especially for patients who haven't had prior systemic (whole-body) anti-cancer therapy. * The New Approach: This study combines two types of drugs: * Envafolimab: An "immunotherapy" drug given as an injection under the skin (subcutaneous). It works by blocking a protein called PD-L1 on cancer cells or immune cells. Blocking PD-L1 helps the patient's own immune system recognize and attack the cancer cells more effectively. * Axitinib: A "targeted therapy" drug taken as a pill twice daily. It works by blocking signals (VEGF receptors) that cancer cells use to grow new blood vessels, essentially starving the tumor of its blood supply. * The Goal: To find out if giving Envafolimab and Axitinib together is safe and effective as the first treatment for patients with advanced kidney cancer. Researchers want to see how well the combination shrinks tumors, controls the cancer, and how long patients live without their cancer getting worse, while carefully monitoring side effects. * Study Design: * Phase II: This is an early stage of testing in patients, focusing mainly on how well the treatment works and its safety profile in a specific group. * Single-Arm: All participants in this study will receive the same combination treatment (Envafolimab + Axitinib). There is no separate group receiving a different treatment or placebo for comparison in this particular study. * Single-Center: Currently being run at Peking Union Medical College Hospital (but could potentially expand). * Participants: Plans to enroll about 30 patients. * Treatment Duration: Patients receive treatment as long as it's working (tumor isn't growing) and they can tolerate the side effects, for up to a maximum of 2 years. 2. Key Information for Patients \& Families: * Who Might Qualify? You may be eligible to participate if you: * Are between 18 and 75 years old. * Have been diagnosed with advanced clear cell kidney cancer (unresectable or metastatic). * Have NOT received any prior systemic anti-cancer treatment (like chemotherapy, immunotherapy, or other targeted therapies) for your advanced kidney cancer. * Have tumors that can be measured on scans (CT or MRI). * Are relatively active and able to care for yourself (ECOG performance status 0 or 1: meaning you are either fully active or restricted in physically strenuous activity but ambulatory and able to do light work). * Have adequate organ function (healthy enough bone marrow, liver, kidneys, heart) as determined by blood tests. * Are expected to live at least 6 more months. * Understand the study and agree to follow the procedures and attend all visits. * Who Would Not Qualify? You would likely not be eligible if you: * Have another active cancer besides the kidney cancer being studied. * Have had previous systemic treatment for your advanced kidney cancer. * Have known severe allergies to similar drugs or components of Envafolimab/Axitinib. * Have an active autoimmune disease needing strong medication (like high-dose steroids or immunosuppressants) within the last 2 years. (Hormone replacements like thyroid meds are okay). * Are taking high-dose steroids (except inhaled/nasal) or other immune-suppressing drugs shortly before starting. * Are using traditional Chinese medicine or immunomodulators within 2 weeks before joining. * Have serious uncontrolled heart problems (like recent heart attack, severe heart failure, unstable angina, significant irregular heartbeats). * Have uncontrolled fluid build-up needing drainage (like large amounts of ascites or pleural effusion). * Are pregnant, breastfeeding, or unwilling to use highly effective contraception during the study and for 6 months after. * Have significant psychiatric, substance abuse, or other medical/social issues that the research team believes would interfere with the study.

All Clinical Trials for AXITINIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00071006 ↗	AG-013736 (Axitinib) In Patients With Poor Prognosis Acute Myeloid Leukemia (AML) Or Myelodysplastic Syndrome (MDS)	Completed	Pfizer	Phase 2	2003-09-01	The study tests the safety and efficacy of axitinib in patients who have the hematologic disease of Acute Myeloid Leukemia or Myelodysplastic Syndrome. The study tests patients who have poor prognosis before entering the study.
NCT00094094 ↗	Anti-angiogenesis Agent AG-013736 in Patients With Advanced Non-Small Cell Lung Cancer	Completed	Pfizer	Phase 2	2005-02-01	This is a Phase 2 study being conducted at multiple centers in the United States and Germany. Patients having non-small cell lung cancer that has spread to other parts of the body (i.e., metastatic) or is locally advanced (i.e., Stage IIIB with malignant pleural effusion) are eligible to participate. Patients must have disease that has been treated with at least 1 prior treatment for metastatic disease (prior adjuvant treatment for localized disease does not count as prior treatment for metastatic disease). The purpose of the study is to test whether the angiogenesis inhibitor AG-013736 is an effective treatment for advanced non-small cell lung cancer as shown by the number of patients in the study who experience significant and durable tumor shrinkage
NCT00219557 ↗	AG-013736 In Combination With Gemcitabine Versus Gemcitabine Alone For Patients With Metastatic Pancreatic Cancer	Completed	Pfizer	Phase 2	2005-07-05	This is a Phase 2 study being conducted at multiple centers in the United States, Europe and Canada. Patients having pancreatic cancer that is locally advanced or that has spread to other parts of the body (i.e., metastatic) are eligible to participate. Patients must have not had any prior systemic treatment for advanced disease. The purpose of the study is to test whether the angiogenesis inhibitor Axitinib [AG-013736] in combination with gemcitabine is an effective treatment for advanced pancreatic cancer vs. gemcitabine alone by overall survival.
NCT00447005 ↗	Study Of AG-013736 (Axitinib) In Patients With Advanced Solid Tumors	Completed	Pfizer	Phase 1	2007-02-01	To evaluate the clinically recommended dose of AG-013736 (Axitinib) in Japanese patients by reviewing the safety of AG-013736 (Axitinib) following single and multiple dosing.
NCT00471146 ↗	Study Of Gemcitabine Plus AG-013736 Versus Gemcitabine For Advanced Pancreatic Cancer.	Completed	Pfizer	Phase 3	2007-07-01	The purpose of this study is to determine whether investigational study drug, AG-013736, and gemcitabine are effective in the first-line treatment of advanced pancreatic cancer.
NCT00569946 ↗	Study Of AG-013736 (Axitinib) As Second-Line Treatment In Patients With Metastatic Renal Cell Cancer (mRCC)	Completed	Pfizer	Phase 2	2007-12-12	To investigate objective tumor response of AG-013736 for metastatic Renal Cell Cancer (mRCC)

Clinical Trial Conditions for AXITINIB

Condition Name

Condition Name for AXITINIB
Intervention	Trials
Renal Cell Carcinoma	19
Clear Cell Renal Cell Carcinoma	12
Metastatic Renal Cell Carcinoma	11
Carcinoma, Renal Cell	7

Condition MeSH

Condition MeSH for AXITINIB
Intervention	Trials
Carcinoma, Renal Cell	73
Carcinoma	59
Kidney Neoplasms	17
Neoplasms	12

Clinical Trial Locations for AXITINIB

Trials by Country

Trials by Country for AXITINIB
Location	Trials
United States	551
Japan	66
China	44
United Kingdom	39
Canada	34

Trials by US State

Trials by US State for AXITINIB
Location	Trials
California	34
Texas	33
New York	28
Ohio	26
Florida	25

Clinical Trial Progress for AXITINIB

Clinical Trial Phase

Clinical Trial Phase for AXITINIB
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	3
PHASE2	10

Clinical Trial Status

Clinical Trial Status for AXITINIB
Clinical Trial Phase	Trials
Completed	60
Recruiting	49
Not yet recruiting	23

Clinical Trial Sponsors for AXITINIB

Sponsor Name

Sponsor Name for AXITINIB
Sponsor	Trials
Pfizer	77
National Cancer Institute (NCI)	14
Merck Sharp & Dohme Corp.	8

Sponsor Type

Sponsor Type for AXITINIB
Sponsor	Trials
Other	150
Industry	142
NIH	14

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

Last updated: October 30, 2025

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Introduction

Axitinib, marketed under the brand name Inlyta, is an oral tyrosine kinase inhibitor primarily targeting vascular endothelial growth factor receptors (VEGFRs). Developed by Pfizer, it has established a significant presence in the treatment landscape for certain cancers, notably advanced renal cell carcinoma (RCC). This article offers a comprehensive update on Axitinib’s clinical trial developments, analyzes current market trends, and projects future market trajectories based on recent data and emerging research.

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Clinical Trials Update for Axitinib

Recent Trial Outcomes and Ongoing Studies

Since its FDA approval in 2012 for advanced RCC, Axitinib’s clinical development has expanded into several indications and combination therapies. Pfizer continues to evaluate its efficacy across multiple tumor types through ongoing clinical trials.

• Renal Cell Carcinoma (RCC):
  The pivotal phase III AXIS trial (2013) demonstrated superior progression-free survival (PFS) with Axitinib versus Sorafenib in previously treated RCC patients,cementing its standard of care. Follow-up studies and post-marketing surveillance continue to affirm its safety profile and efficacy.

• Combination Therapy Trials:
  Recent trials explore Axitinib combined with immune checkpoint inhibitors such as Pembrolizumab and Avelumab. The KEYNOTE-426 (NCT02853331) phase III trial evaluating Axitinib plus Pembrolizumab in first-line metastatic RCC showed significant improvements in overall response rate (ORR) and PFS, prompting regulatory review extensions.

• Other Cancer Indications:
  Ongoing trials investigate Axitinib in hepatocellular carcinoma (HCC) (NCT02299999), thyroid cancers, and certain soft tissue sarcomas. Results from early-phase studies indicate promising activity, though regulatory approvals are pending or in negotiation.

Key Trials and Outcomes

• GETUG-Ax (NCT03786251):
  Exploring Axitinib in advanced ovarian cancer, with preliminary data indicating disease stabilization.

• Safety Profile:
  Consistent across trials, with manageable hypertension, diarrhea, and fatigue as common adverse events; serious adverse events are relatively infrequent, supporting its tolerability.

Regulatory Status and Future Approvals

While Axitinib holds approval in RCC globally, regulatory agencies are reviewing data for expanded indications:

• European Medicines Agency (EMA): Anticipated approval extensions based on recent combination studies.
• FDA: Potential label updates for combination therapy indications, pending ongoing trial results.

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Market Analysis for Axitinib

Current Market Landscape

The global oncology drug market exceeds $160 billion annually and continues to grow, driven by rising cancer incidence and innovation in targeted therapies. Axitinib occupies a lucrative niche within anti-angiogenic agents for RCC and other indications.

• Market Share:
  In the RCC segment, Axitinib competes with Sorafenib, Sunitinib, and Pazopanib. While Sunitinib remains the market leader, Axitinib has carved a substantial share due to its favorable efficacy profile and tolerability in second-line settings.

• Revenue Contribution:
  Pfizer’s oncology portfolio reports revenues of approximately $20 billion annually, with Axitinib contributing a significant portion—estimated at $1-2 billion worldwide (pre-pandemic figures). The recent uptick in combination therapy trials suggests potential revenue expansion.

• Regional Dynamics:
  North America and Europe account for the majority of sales, with Asia-Pacific emerging rapidly—fueled by increasing RCC diagnoses and expanding healthcare infrastructure.

Market Drivers

• Unmet Medical Needs:
  Growing prevalence of RCC, coupled with the demand for better tolerated therapies, sustains Axitinib’s relevance.

• Combination Strategies:
  The push toward immunotherapy combinations enhances Axitinib’s value proposition, fostering pipeline growth.

• Regulatory Approvals:
  Pending approvals for new indications will broaden its application scope, likely elevating market penetration.

Market Challenges

• Competition:
  With new agents like Lenvatinib and multi-kinase inhibitors entering the field, maintaining market share necessitates differentiation through efficacy and safety.

• Cost Pressures:
  As with many targeted therapies, pricing strategies and reimbursement landscapes influence market access.

• Patent Expiry and Biosimilars:
  Although patent protection remains strong, imminent patent cliffs could affect pricing and market dynamics.

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Future Market Projections

Forecasting Based on Clinical and Market Trends

Market analysis forecasts suggest steady growth for Axitinib over the next five years, with compound annual growth rates (CAGRs) estimated at 5-8%, driven predominantly by the expansion of its indications and combination regimens.

• 2023-2028 Revenue Estimates:
  Projected revenues could reach $2.5–3 billion, contingent upon successful regulatory approvals, uptake of combination therapies, and increased adoption in emerging markets.

• Growth Catalysts:

  • Approval for first-line combination with Pembrolizumab in RCC (anticipated in 2023).
  • Positive trial outcomes in other cancers such as HCC and thyroid carcinomas.
  • Enhanced awareness and screening programs in Asia-Pacific.

• Market Risks:

  • Competition from PD-1/PD-L1 inhibitors with standalone efficacy.
  • Potential safety concerns or regulatory delays.
  • Pricing pressures and healthcare reforms.

Implications for Stakeholders

Pharmaceutical companies, investors, and healthcare providers should monitor ongoing trial outcomes and regulatory decisions closely. Capitalizing on Axitinib’s potential hinges upon strategic positioning, pricing models, and understanding regional deployment challenges.

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Key Takeaways

• Axitinib remains a cornerstone targeted therapy for advanced RCC, with ongoing clinical trials expanding its therapeutic frontier.
• Combination therapies involving Axitinib and immunotherapies are showing promising efficacy, poised to reshape its market potential.
• The global market for Axitinib is poised for sustained growth, driven by evolving treatment paradigms, new indications, and regional expansion.
• Competition, cost considerations, and regulatory landscapes will influence its market trajectory.
• Stakeholders should anticipate regulatory announcements and emerging clinical data to optimize commercial and clinical strategies.

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FAQs

Q1: What are the primary indications for Axitinib currently?
A1: Axitinib is approved mainly for advanced or metastatic renal cell carcinoma (RCC), particularly after failure of prior systemic therapy.

Q2: How effective is Axitinib compared to other VEGFR inhibitors?
A2: Clinical trials demonstrate comparable efficacy with a more favorable tolerability profile, particularly in second-line RCC settings.

Q3: Are there ongoing efforts to expand Axitinib’s indications?
A3: Yes, multiple trials are investigating its use in hepatocellular carcinoma, thyroid cancers, and combination therapies for various tumor types.

Q4: What are the main safety concerns associated with Axitinib?
A4: Common adverse events include hypertension, diarrhea, fatigue, and hand-foot syndrome; serious adverse events are less frequent but include cardiovascular complications.

Q5: When might Axitinib’s market share increase significantly?
A5: Its market share is likely to grow with positive results from combination therapy trials and approvals for new indications, especially in the first-line setting.

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References

1. (1) European Medicines Agency. Inlyta (Axitinib) Summary of Product Characteristics.
2. (2) Novartis. “Axitinib (Inlyta) in Renal Cell Carcinoma,” Clinical Trial Data, 2013.
3. (3) Pfizer. “Axitinib Clinical Trials,” 2022.
4. (4) MarketWatch. “Oncology Drugs Market Size & Share,” 2022.
5. (5) ClinicalTrials.gov. “Ongoing studies involving Axitinib,” 2023.

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