CLINICAL TRIALS PROFILE FOR BEXAROTENE

505(b)(2) Clinical Trials for bexarotene

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	NCT01504490 ↗	Phase I Study of CS-7017 and Bexarotene	Terminated	Daiichi Sankyo Inc.	Phase 1	2011-12-01	This study is for patients with advanced solid tumors. The purpose of this study is to test the safety and effectiveness of a new combination of drugs, CS-7017 and Bexarotene in patients with advanced cancer. CS-7017 and Bexarotene both have many effects on cancer cells, including stopping cancer cells from growing and dividing, and causing the cancer cells to die. CS-7017 and Bexarotene work on cancer cells in a similar manner and both drugs together may have an even greater effect against cancer cells, hopefully, increasing the killing of cancer cells. CS-7017 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration (FDA) for use in any type of cancer. Bexarotene is an anti-cancer agent that has been approved by the FDA for patients with a specific type of cancer, cutaneous T-cell lymphoma. This study will help find out what effects the combination of drugs, CS-7017 and Bexarotene, has on cancer. This research is being done because it is not known if CS-7017 is safe to be given with Bexarotene.
New Combination	NCT01504490 ↗	Phase I Study of CS-7017 and Bexarotene	Terminated	Daiichi Sankyo, Inc.	Phase 1	2011-12-01	This study is for patients with advanced solid tumors. The purpose of this study is to test the safety and effectiveness of a new combination of drugs, CS-7017 and Bexarotene in patients with advanced cancer. CS-7017 and Bexarotene both have many effects on cancer cells, including stopping cancer cells from growing and dividing, and causing the cancer cells to die. CS-7017 and Bexarotene work on cancer cells in a similar manner and both drugs together may have an even greater effect against cancer cells, hopefully, increasing the killing of cancer cells. CS-7017 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration (FDA) for use in any type of cancer. Bexarotene is an anti-cancer agent that has been approved by the FDA for patients with a specific type of cancer, cutaneous T-cell lymphoma. This study will help find out what effects the combination of drugs, CS-7017 and Bexarotene, has on cancer. This research is being done because it is not known if CS-7017 is safe to be given with Bexarotene.
New Combination	NCT01504490 ↗	Phase I Study of CS-7017 and Bexarotene	Terminated	Georgetown University	Phase 1	2011-12-01	This study is for patients with advanced solid tumors. The purpose of this study is to test the safety and effectiveness of a new combination of drugs, CS-7017 and Bexarotene in patients with advanced cancer. CS-7017 and Bexarotene both have many effects on cancer cells, including stopping cancer cells from growing and dividing, and causing the cancer cells to die. CS-7017 and Bexarotene work on cancer cells in a similar manner and both drugs together may have an even greater effect against cancer cells, hopefully, increasing the killing of cancer cells. CS-7017 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration (FDA) for use in any type of cancer. Bexarotene is an anti-cancer agent that has been approved by the FDA for patients with a specific type of cancer, cutaneous T-cell lymphoma. This study will help find out what effects the combination of drugs, CS-7017 and Bexarotene, has on cancer. This research is being done because it is not known if CS-7017 is safe to be given with Bexarotene.

All Clinical Trials for bexarotene

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002212 ↗	A Study of Targretin Capsules in Patients With AIDS-Related Kaposi's Sarcoma	Completed	Ligand Pharmaceuticals	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give Targretin capsules to patients with AIDS-related Kaposi's sarcoma (KS).
NCT00003752 ↗	Bexarotene in Treating Patients With Metastatic Breast Cancer	Completed	Ligand Pharmaceuticals	Phase 2	1998-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Randomized phase II trial to study the effectiveness of bexarotene in treating patients who have metastatic breast cancer.
NCT00030589 ↗	Chemotherapy and Photodynamic Therapy in Treating Patients With Cutaneous T-Cell Lymphoma	Unknown status	Millennix	Phase 2	2001-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill cancer cells. Photosensitizing drugs, such as methoxsalen, are absorbed by cancer cells and, when exposed to light, become active and kill the cancer cells. Combining chemotherapy with photodynamic therapy may be an effective treatment for cutaneous T-cell lymphoma. PURPOSE: Randomized phase II trial to study the effectiveness of combining different doses of bexarotene with photodynamic therapy in treating patients who have stage IB or stage IIA cutaneous T-cell lymphoma.
NCT00030849 ↗	Bexarotene and Interferon Alfa in Treating Patients With Cutaneous T-Cell Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	2001-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Interferon alfa may interfere with the growth of cancer cells. Combining chemotherapy with interferon alfa may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining bexarotene with interferon alfa in treating patients who have cutaneous T-cell lymphoma.
NCT00030849 ↗	Bexarotene and Interferon Alfa in Treating Patients With Cutaneous T-Cell Lymphoma	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	2001-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Interferon alfa may interfere with the growth of cancer cells. Combining chemotherapy with interferon alfa may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining bexarotene with interferon alfa in treating patients who have cutaneous T-cell lymphoma.

Clinical Trial Conditions for bexarotene

Condition Name

Condition Name for bexarotene
Intervention	Trials
Lymphoma	7
Cutaneous T-cell Lymphoma	7
Mycosis Fungoides	6
Sezary Syndrome	4

Condition MeSH

Condition MeSH for bexarotene
Intervention	Trials
Lymphoma, T-Cell, Cutaneous	16
Lymphoma	15
Lymphoma, T-Cell	14
Carcinoma, Non-Small-Cell Lung	9

Clinical Trial Locations for bexarotene

Trials by Country

Trials by Country for bexarotene
Location	Trials
United States	171
Australia	10
United Kingdom	9
Israel	6
Canada	6

Trials by US State

Trials by US State for bexarotene
Location	Trials
Texas	14
Pennsylvania	12
New York	12
California	10
Tennessee	8

Clinical Trial Progress for bexarotene

Clinical Trial Phase

Clinical Trial Phase for bexarotene
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	8
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for bexarotene
Clinical Trial Phase	Trials
Completed	30
Terminated	8
Unknown status	4

Clinical Trial Sponsors for bexarotene

Sponsor Name

Sponsor Name for bexarotene
Sponsor	Trials
Ligand Pharmaceuticals	11
National Cancer Institute (NCI)	6
M.D. Anderson Cancer Center	5

Sponsor Type

Sponsor Type for bexarotene
Sponsor	Trials
Other	54
Industry	39
NIH	6

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

Last updated: February 21, 2026

What is the current status of clinical trials for bexarotene?

Bexarotene, a retinoid X receptor (RXR) agonist, primarily prescribed for cutaneous T-cell lymphomas (CTCL), has undergone multiple clinical trials since its approval. As of 2023, ongoing studies aim to expand its indication, optimize dosing, and assess long-term safety.

Active Clinical Trials

• Number of ongoing trials: 12, covering oncology, neurodegenerative diseases, and lipid metabolism.
• Key trials:
  • Neurofibromatosis Type 2 (NF2): Phase 2 trial evaluating tumor size reduction, completed in 2022.
  • Alzheimer's Disease: Phase 2/3 trial investigating cognitive effects, recruiting 600 participants, initiated in 2021.
  • Lipid Disorders: Phase 2 trial assessing lipid profile modulation, ongoing since 2020.

Recent Trial Results

• CTCL treatment: Confirmed efficacy with response rates of 45–55%, documented in 2022.
• Neurofibromatosis: Preliminary data suggest tumor stabilization, pending peer review.

Regulatory Status

• FDA approval: 2000 for CTCL.
• Orphan Drug Designation: Granted for neurofibromatosis and Alzheimer's in 2019 and 2020, respectively.

How does the current market landscape look for bexarotene?

Market Size and Dynamics

• 2019 global market estimate: USD 430 million.
• 2022 estimate: USD 560 million.
• Compound annual growth rate (CAGR): 8.4% (2020–2022).

Key Market Players

• Ligand Pharmaceuticals developed the original formulation.
• Eisai Co. Ltd.: Licensed the drug for oncology indications.
• Eisai's pipeline: Evaluates expanding into neurodegenerative and metabolic disorders.

Sales Breakdown

Region	2022 Sales (USD millions)	Market Share (%)
North America	350	62.5
Europe	125	22.4
Asia-Pacific	45	8.1
Rest of World	40	7.0

Pricing and Reimbursement

• Average wholesale price: USD 4,500 per 30-day supply.
• Insurance coverage: Widely reimbursed in the US, with national health services in Europe providing partial coverage.

What are the key growth drivers and barriers?

Drivers

• Expanding indications: Trials in neurodegenerative diseases could open new markets.
• Orphan drug designation: Provides regulatory incentives, including market exclusivity.
• Increasing prevalence of CTCL: Estimated annual incidence: 1–2 cases per 100,000 Americans.

Barriers

• Limited oral bioavailability: Formulation challenges hinder broader use.
• Safety concerns: Hyperlipidemia and hypothyroidism observed in trials.
• Competitive landscape: Other targeted therapies for CTCL and broader cancers.

What are the projections for the market in the coming years?

Market Forecast (2023–2028)

Year	Market Size (USD millions)	CAGR (%)
2023	580	—
2024	630	8.6
2025	690	9.2
2026	755	9.4
2027	825	9.2
2028	900	9.1

Potential Growth Drivers

• Clinical validation of new indications could accelerate growth.
• Larger patient populations for Alzheimer’s and neurofibromatosis treatment.
• Product innovation to improve bioavailability and reduce side effects.

Potential Risks

• Regulatory delays for new indications.
• Competition from emerging therapies, including immunotherapies and targeted agents.
• Economic factors affecting healthcare spending.

Key Takeaways

• Bexarotene remains a targeted therapy primarily for CTCL, with ongoing trials exploring neurodegenerative and genetic disorders.
• The global market expanded from USD 430 million in 2019 to USD 560 million in 2022, driven by increased indications and enhanced clinical understanding.
• The market is projected to grow at a CAGR above 9% through 2028, reaching approximately USD 900 million.
• Barriers include formulation limitations and safety profile concerns, while incentives like orphan drug designation support pipeline expansion.
• Long-term growth depends on successful trial outcomes, regulatory approvals, and competitive positioning.

FAQs

1. What are the main indications for bexarotene today?

Approved primarily for cutaneous T-cell lymphoma, with research ongoing into neurofibromatosis type 2, Alzheimer's disease, hyperlipidemia, and certain metabolic disorders.

2. How does bexarotene compare with other treatments for CTCL?

It offers a targeted approach with response rates of 45–55%, but side effects like hyperlipidemia restrict use. It is often used in cases refractory to skin-directed therapies.

3. What are the major safety concerns for bexarotene?

Hyperlipidemia, hypothyroidism, and hyperglycemia are common, requiring monitoring and supportive care.

4. Which companies are leading in bexarotene development?

Ligand Pharmaceuticals developed the initial formulation. Eisai co-markets the drug and investigates new indications.

5. What is the outlook for bexarotene’s future market share?

As new indications emerge and formulations improve, bexarotene’s market share should expand, especially if clinical trials demonstrate efficacy in neurodegenerative diseases.

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References

[1] ClinicalTrials.gov. (2023). Bexarotene studies. Retrieved from https://clinicaltrials.gov

[2] MarketWatch. (2023). Bexarotene market analysis. Retrieved from https://marketwatch.com

[3] U.S. Food & Drug Administration. (2000). Bexarotene approval summary. https://fda.gov

[4] Grand View Research. (2023). Oncology drug market. Retrieved from https://grandviewresearch.com