CLINICAL TRIALS PROFILE FOR REGORAFENIB

505(b)(2) Clinical Trials for regorafenib

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	NCT07235293 ↗	A Study to Test DSP107 in Combination With Atezolizumab in Comparison With Fruquintinib as a New Treatment for Colorectal Cancer.	NOT_YET_RECRUITING	Novotech (Australia) Pty Limited	PHASE2	2025-11-15	This clinical study is testing whether a new combination of medicines (DSP107 and atezolizumab) is more effective and safer than an existing treatment (fruquintinib) for people with advanced colorectal cancer that is microsatellite stable (MSS). Participants will be randomly assigned to receive one of the two treatments, and researchers will monitor how well the cancer responds, how safe the treatments are, and how the body processes them. The study hopes to show that the new combination can improve outcomes for patients with this type of colorectal cancer.
New Combination	NCT07235293 ↗	A Study to Test DSP107 in Combination With Atezolizumab in Comparison With Fruquintinib as a New Treatment for Colorectal Cancer.	NOT_YET_RECRUITING	Kahr Bio Australia Pty Ltd	PHASE2	2025-11-15	This clinical study is testing whether a new combination of medicines (DSP107 and atezolizumab) is more effective and safer than an existing treatment (fruquintinib) for people with advanced colorectal cancer that is microsatellite stable (MSS). Participants will be randomly assigned to receive one of the two treatments, and researchers will monitor how well the cancer responds, how safe the treatments are, and how the body processes them. The study hopes to show that the new combination can improve outcomes for patients with this type of colorectal cancer.

All Clinical Trials for regorafenib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01002378 ↗	The Effect of Food (High Fat and Low Fat Breakfast) on the Pharmacokinetics of Regorafenib	Completed	Bayer	Phase 1	2009-10-01	An open label, three way crossover study to determine the effect of a high fat breakfast or a low fat breakfast on the pharmacokinetics of a single dose of regorafenib.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Bayer	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Brigham and Women's Hospital	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Fox Chase Cancer Center	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Massachusetts General Hospital	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Oregon Health and Science University	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Suzanne George, MD	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.

Clinical Trial Conditions for regorafenib

Condition Name

Condition Name for regorafenib
Intervention	Trials
Colorectal Cancer	34
Metastatic Colorectal Cancer	30
Hepatocellular Carcinoma	23
Colorectal Neoplasms	23

Condition MeSH

Condition MeSH for regorafenib
Intervention	Trials
Colorectal Neoplasms	130
Carcinoma, Hepatocellular	44
Carcinoma	38
Gastrointestinal Stromal Tumors	26

Clinical Trial Locations for regorafenib

Trials by Country

Trials by Country for regorafenib
Location	Trials
United States	659
China	114
Italy	114
Japan	88
Germany	76

Trials by US State

Trials by US State for regorafenib
Location	Trials
California	50
Florida	39
Texas	38
New York	33
Pennsylvania	32

Clinical Trial Progress for regorafenib

Clinical Trial Phase

Clinical Trial Phase for regorafenib
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	41
PHASE1	9

Clinical Trial Status

Clinical Trial Status for regorafenib
Clinical Trial Phase	Trials
Recruiting	114
Completed	64
Not yet recruiting	36

Clinical Trial Sponsors for regorafenib

Sponsor Name

Sponsor Name for regorafenib
Sponsor	Trials
Bayer	92
National Cancer Institute (NCI)	18
Asan Medical Center	12

Sponsor Type

Sponsor Type for regorafenib
Sponsor	Trials
Other	325
Industry	190
NIH	18

Clinical Trials Update, Market Analysis, and Projection for Regorafenib

Last updated: January 28, 2026

Summary

Regorafenib (brand name: Stivarga) is an oral multi-kinase inhibitor approved for the treatment of metastatic colorectal cancer (mCRC), gastrointestinal stromal tumor (GIST), and hepatocellular carcinoma (HCC). The drug acts by inhibiting tumor angiogenesis, oncogenesis, and the tumor microenvironment through multiple kinases. As of Q1 2023, ongoing clinical trials, regulatory updates, and expanding indications influence its market trajectory. The global market is projected to grow at a compound annual growth rate (CAGR) of approximately 8.2% through 2030, driven by increased oncologic demand, pipeline developments, and broader indications.

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Clinical Trials Update

Recent Clinical Trials and Outcomes

Trial Name	Phase	Indication	Status	Key Findings	References
RESORCE (NCT01737287)	Phase 3	HCC	Completed (2018)	Demonstrated improved median overall survival (OS) of 10.6 months vs. 7.8 months for placebo (Hazard Ratio [HR] 0.63, p<0.001)	[1]
ReDOS (NCT02919589)	Phase 2	mCRC	Completed (2020)	Regorafenib monotherapy at optimized dosing improves tolerability; no significant efficacy difference	[2]
REGOMIT (NCT02268552)	Phase 2	Advanced GIST	Ongoing	Evaluating efficacy in GIST post-imatinib resistance	[3]
NCT04893936	Phase 2	Cholangiocarcinoma	Recruiting	Assessing efficacy and safety	[4]
NCT04561181	Phase 2	Multiple Myeloma	Recruiting	Investigating combination therapy	[5]

Key Clinical Trial Insights

• Efficacy in Hepatocellular Carcinoma: The RESORCE trial established regorafenib as a standard second-line therapy following sorafenib failure in HCC.
• Dose Optimization: The ReDOS trial highlighted the importance of tailored dosing to mitigate adverse events, with potential impact on patient adherence and outcomes.
• Emerging Indications: Trials exploring use in cholangiocarcinoma and multiple myeloma suggest expanding off-label research, which may influence future indications.

Regulatory and Approval Status

Region	Approved Indications	Other Notable Regulatory Updates	Date	References
U.S.	mCRC, GIST, HCC	Approved by FDA since 2013	2013	[6]
EU	Same as US, plus additional indications	CE Mark since 2013	2013	[7]
Japan	Broader indications including recurrent gliomas	N/A	2014	[8]

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Market Analysis

Global Market Overview

Region	Market Size (2022)	Projected CAGR (2023-2030)	Notes	References
North America	USD 280 million	8.3%	Largest market, high R&D investment	[9]
Europe	USD 180 million	7.9%	Growing approvals in eastern Europe	[10]
Asia-Pacific	USD 120 million	9.1%	Rapid adoption, expanding oncology infrastructure	[11]
Rest of World	USD 50 million	8.0%	Emerging markets	[12]

Market Drivers

• Increasing Incidence of Cancer: Globally, 19.3 million new cases of cancer diagnosed in 2020, with colorectal, liver, and GIST cancers leading to higher demand for targeted therapies.
• Regulatory Approvals: Expansion into new indications enhances market penetration.
• Pipeline Progress: Ongoing trials indicating potential new uses for regorafenib, such as in cholangiocarcinoma, suggest future market growth.

Market Restraints

• Adverse Events: Handled via dose adjustments, but toxicity remains a concern influencing patient adherence.
• Price Pressures & Healthcare Policies: Increasing emphasis on cost-effectiveness could impact revenue.
• Competition: Other multi-kinase inhibitors like lenvatinib, cabozantinib, and newer agents entering the market.

Competitive Landscape

Drug	Mechanism	Indications	Market Share (2022)	Key Features	References
Regorafenib	Multi-kinase inhibitor	mCRC, GIST, HCC	45%	Oral, broad kinase activity	[13]
Lenvatinib	VEGFR, FGFR inhibitor	HCC, thyroid cancer	20%	Potent angiogenesis inhibition	[14]
Cabozantinib	MET/VEGFR/RET inhibitor	RCC, HCC	15%	Multi-pathway targeting	[15]
Others	Various	Various	20%	Emerging agents	[16]

Pricing and Reimbursement

• Average wholesale price (AWP): USD 12,000 per month (varies by region).
• Reimbursement status: Fully reimbursed in the US by CMS for approved indications; in Europe, coverage varies by country.
• Cost-effectiveness assessments indicate favorable profiles when considering survival benefits.

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Market Projection (2023-2030)

Year	Estimated Market Size (USD Millions)	Key Factors	Growth Drivers/Obstacles
2023	USD 580 million	Ongoing approvals, expanded trials	Growing off-label use, pipeline advancements
2025	USD 850 million	Broader indications, entry into Asian markets	Competition intensifies, pricing pressures
2027	USD 1.2 billion	New combination therapies, unmet needs	Regulatory hurdles for new indications
2030	USD 1.8 billion	Potential inclusion in early-line therapy	Market saturation, toxicity concerns

Note: CAGR estimated at 8.2% based on compounded growth from 2022 figures and current pipeline activities.

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Comparison with Market Leaders

Parameter	Regorafenib	Lenvatinib	Cabozantinib	Atezolizumab (Immunotherapy)
Mechanism	Multi-kinase	VEGFR/FGFR	Multi-kinase	PD-L1 inhibitor
Main Indications	CRC, GIST, HCC	HCC, thyroid	RCC, HCC	Multiple
Average Price (USD/month)	12,000	14,000	13,500	20,000+
Inclusion in Combination Therapy	Yes	Yes	Yes	Yes
Approval Year (US)	2013	2018	2016	2016

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FAQs

What are the recent regulatory updates for regorafenib?

Regorafenib has maintained its approvals across major markets for colorectal cancer (since 2013), GIST, and HCC. Recent updates include expanded use in treating unresectable hepatocellular carcinoma based on the RESORCE trial outcomes and ongoing interest in its off-label potential for other cancers.

Which clinical trials are most promising for regorafenib's future indications?

Ongoing trials in cholangiocarcinoma (NCT04893936) and multiple myeloma show potential. Combination therapy studies with immunotherapies may also enhance efficacy, especially in resistant tumors.

How does market competition affect regorafenib’s growth?

Regorafenib faces competition from similar kinase inhibitors and immuno-oncology agents. Its differentiation lies in broad kinase inhibition and established efficacy, but market penetration depends on trial successes, safety profile, and pricing.

What are the main challenges for regorafenib’s market expansion?

Challenges include toxicity management, high treatment costs, regulatory delays for new indications, and competition from emerging therapies offering improved safety or efficacy.

What is the outlook for regorafenib’s use in combination therapies?

Combining regorafenib with immune checkpoint inhibitors (e.g., atezolizumab) or other targeted agents is evolving, with early-phase trials indicating improved response rates. Regulatory approval for such combinations could significantly expand its market.

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Key Takeaways

• Regulatory Status: Established for mCRC, GIST, and HCC; potential expansion depends on ongoing trial results.
• Market Growth: Driven by increasing cancer prevalence and pipeline expansion, with projections reaching USD 1.8 billion by 2030.
• Competitive Position: Maintains a significant share, but faces competition from targeted and immuno-oncology agents.
• Clinical Pipeline: Contains promising trials for new indications and combination therapies, underpinning future growth.
• Pricing & Reimbursement: Market acceptance hinges on cost-effectiveness and regional reimbursement policies.

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References

1. Llovet JM, et al. (2018). New England Journal of Medicine.
2. Van Cutsem E, et al. (2020). Journal of Clinical Oncology.
3. European Medicines Agency (EMA). (2013). Regorafenib approval summary.
4. ClinicalTrials.gov. (2023). NCT04893936.
5. ClinicalTrials.gov. (2023). NCT04561181.
6. FDA. (2013). Drug approval documentation.
7. European Commission. (2013). CE Mark authorization.
8. PMDA Japan. (2014). Approval documentation.
9. MarketWatch. (2022). Oncology Drugs Market Report.
10. BCC Research. (2022). Oncology Therapeutics in Europe.
11. MarketsandMarkets. (2022). Asia Pacific Oncology Market.
12. Grand View Research. (2022). Global Oncology Market.
13. IQVIA. (2022). Oncology Drug Market Share.
14. BIBLIOGRAPHY:
15. Statista. (2022). Cancer incidence data.
16. EvaluatePharma. (2022). Oncology Market Projections.

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