CLINICAL TRIALS PROFILE FOR VESANOID

All Clinical Trials for VESANOID

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00005825 ↗	Combination Chemotherapy in Treating Patients With Stage IIIB or Stage IV Non-small Cell Lung Cancer	Unknown status	Raghu Nandan, M.D., Inc	Phase 2	1998-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have stage IIIB or stage IV non-small cell lung cancer.
NCT00006220 ↗	Arsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy	Terminated	National Cancer Institute (NCI)	Phase 1/Phase 2	1999-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. PURPOSE: Phase I/II trial to study the effectiveness of arsenic trioxide with or without tretinoin in treating patients who have hematologic cancer that has not responded to previous therapy.
NCT00006220 ↗	Arsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy	Terminated	Washington University School of Medicine	Phase 1/Phase 2	1999-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. PURPOSE: Phase I/II trial to study the effectiveness of arsenic trioxide with or without tretinoin in treating patients who have hematologic cancer that has not responded to previous therapy.
NCT00326170 ↗	Phase II 5-Azacytidine Plus VPA Plus ATRA	Completed	Celgene Corporation	Phase 2	2005-07-01	5-aza is a chemotherapy drug with activity in leukemia and myelodysplastic syndromes (MDS). Researchers hope that valproic acid (VPA) and all-trans retinoic acid (ATRA)will increase the effects of 5-aza. The goal of this clinical research study is to find the highest safe dose of valproic acid (VPA) that can be given in combination with 5-azacytidine (5-aza) and all-trans retinoic acid (ATRA) in the treatment of AML and MDS. The safety and effectiveness of this combination therapy will also be studied. Additional blood and bone marrow samples will be requested. These samples will be used to evaluate the effect of the treatment on leukemic cells. In addition, any leftover blood and bone marrow samples that are collected at the start of the study and during the regularly scheduled evaluations to be sent for research studies. The research studies will examine changes in the blood and bone marrow cells that might help explain the causes of leukemia and MDS and how the combination of 5-aza, VPA, and ATRA works.
NCT00326170 ↗	Phase II 5-Azacytidine Plus VPA Plus ATRA	Completed	M.D. Anderson Cancer Center	Phase 2	2005-07-01	5-aza is a chemotherapy drug with activity in leukemia and myelodysplastic syndromes (MDS). Researchers hope that valproic acid (VPA) and all-trans retinoic acid (ATRA)will increase the effects of 5-aza. The goal of this clinical research study is to find the highest safe dose of valproic acid (VPA) that can be given in combination with 5-azacytidine (5-aza) and all-trans retinoic acid (ATRA) in the treatment of AML and MDS. The safety and effectiveness of this combination therapy will also be studied. Additional blood and bone marrow samples will be requested. These samples will be used to evaluate the effect of the treatment on leukemic cells. In addition, any leftover blood and bone marrow samples that are collected at the start of the study and during the regularly scheduled evaluations to be sent for research studies. The research studies will examine changes in the blood and bone marrow cells that might help explain the causes of leukemia and MDS and how the combination of 5-aza, VPA, and ATRA works.
NCT00339196 ↗	5-azacytidine Valproic Acid and ATRA in AML and High Risk MDS	Completed	Assistance Publique - Hôpitaux de Paris	Phase 2	2006-07-01	MULTICENTERS. Uncontrolled and open phase II study. Evaluation of the effectiveness of a treatment associating 5 Azacytidine,Valproic acid ,Retinoic Acid at subjects-reached of syndromes myelodysplasia and acute MYELOID leukaemia Hematological response at 6 months Uncontrolled prospective cohort.
NCT00617409 ↗	To Immunize Patients With Extensive Stage SCLC Combined With Chemo With or Without All Trans Retinoic Acid	Completed	H. Lee Moffitt Cancer Center and Research Institute	Phase 2	2007-10-02	The purpose of this research study is to test a tumor (cancer) vaccine given along with chemotherapy to determine if this vaccine will increase the chances of the tumor shrinking and/or the amount of time that people who have this disease will live.

Clinical Trial Conditions for VESANOID

Condition Name

Condition Name for VESANOID
Intervention	Trials
Acute Myelogenous Leukemia	3
Myelodysplastic Syndrome	3
Leukemia	2
Acute Myeloid Leukemia	2

Condition MeSH

Condition MeSH for VESANOID
Intervention	Trials
Leukemia	10
Leukemia, Myeloid, Acute	7
Myelodysplastic Syndromes	6
Preleukemia	6

Clinical Trial Locations for VESANOID

Trials by Country

Trials by Country for VESANOID
Location	Trials
United States	69
Canada	6
Australia	6
France	2
New Zealand	2

Trials by US State

Trials by US State for VESANOID
Location	Trials
Texas	6
Oregon	3
California	3
Colorado	3
Ohio	3

Clinical Trial Progress for VESANOID

Clinical Trial Phase

Clinical Trial Phase for VESANOID
Clinical Trial Phase	Trials
PHASE3	1
Phase 3	2
Phase 2	10

Clinical Trial Status

Clinical Trial Status for VESANOID
Clinical Trial Phase	Trials
Completed	8
RECRUITING	6
Active, not recruiting	3

Clinical Trial Sponsors for VESANOID

Sponsor Name

Sponsor Name for VESANOID
Sponsor	Trials
Celgene Corporation	4
M.D. Anderson Cancer Center	4
National Cancer Institute (NCI)	3

Sponsor Type

Sponsor Type for VESANOID
Sponsor	Trials
Other	37
Industry	9
NIH	4

Vesanoid (tretinoin) Clinical Trials Update, Market Analysis, and Exclusivity Outlook

Last updated: July 28, 2026

Vesanoid is the brand name for tretinoin (all-trans retinoic acid, ATRA) in oral capsules used in acute promyelocytic leukemia (APL). Public information available up to mid-2024 is insufficient to produce a reliable, complete “clinical trials update” and forward-looking “market projections” specific to Vesanoid’s current commercial trajectory across geographies and formulations.

What is Vesanoid (tretinoin) approved for and what evidence supports it?

Vesanoid’s core approved indication is acute promyelocytic leukemia (APL), historically based on the differentiation effect of ATRA on promyelocytes. Product labeling and clinical history place Vesanoid within the established ATRA-based standard of care for APL.

What populations and lines of therapy are covered?

Key clinical use cases in APL include:

• Induction therapy with ATRA as part of APL treatment regimens
• Use in combination strategies that may include chemotherapy and/or arsenic-based regimens, depending on risk stratification and era-specific standards

How does ATRA fit with modern APL regimens?

Current APL practice often uses risk-adapted combinations involving:

• ATRA plus arsenic trioxide and/or cytotoxic chemotherapy
• Treatment intensity adjustments based on baseline WBC count and other factors

What clinical trials involve Vesanoid or ATRA today (and which endpoints matter)?

A complete, up-to-date clinical trials map for “Vesanoid” specifically requires a current linkage between the brand and each study record, including sponsor labeling and registry crosswalks. With only the information provided in the prompt, a fully accurate trial-by-trial update cannot be produced.

Which trial endpoints drive APL differentiation therapy decisions?

For APL, trials typically focus on:

• Complete remission (CR) rate
• Relapse-free survival (RFS)
• Event-free survival (EFS)
• Overall survival (OS)
• Molecular remission (where measured)
• Time to differentiation
• Safety endpoints including retinoic acid syndrome, hepatic events, and hematologic toxicity

What are the major formulation and dosing variables?

For ATRA products, differences can include:

• Dose scheduling during induction and consolidation
• Capsule strength availability and excipient-related tolerability
• Regional product variations affecting exposure and administration instructions

What market is Vesanoid in today, and what drives demand for APL ATRA?

Vesanoid demand is tied to APL epidemiology and prescribing of ATRA-based regimens. Commercial dynamics also depend on:

• Availability and price of generic ATRA
• Treatment guideline adoption (ATRA plus arsenic-based regimens vs other combinations)
• Hospital formulary behavior and procurement

Key demand drivers

• Stable incidence of APL within acute leukemia populations
• Continued use of ATRA in standard regimens
• Procurement pressure from generic entrants

Key headwinds

• Price compression from generic tretinoin products
• Substitution risk where dosing schedules and product tolerability are equivalent
• Limited brand-new differentiation opportunities because ATRA is an established agent

How many competitors does Vesanoid face from generic tretinoin, and what does that imply for pricing?

Vesanoid competes primarily against generic tretinoin (ATRA) oral capsule offerings. In mature markets, brand-level revenue typically declines as generics capture a large share through:

• Lower acquisition costs
• Wide therapeutic equivalence assumptions
• Institutional standardization on generics

When does Vesanoid lose exclusivity, and what is the generic or biosimilar entry risk?

Vesanoid is a legacy product. For APL, the main competitive threat is small-molecule generic substitution, not biosimilars.

A reliable exclusivity and patent-loss timeline for “Vesanoid” requires:

• Patent estate mapping to the specific FDA-listed product (strength, dosage form)
• Listing of FDA Orange Book exclusivity and relevant listed patents
• Current litigation or settlement status

With the information available in the prompt alone, a complete exclusivity calendar and generic entry risk assessment cannot be produced.

What is the Orange Book status of Vesanoid?

Orange Book status is determined by:

• Listed patents for the specific NDA product
• Whether patents are still active
• Any periods of exclusivity that remain for relevant strengths

A precise Orange Book listing table with patent numbers and expiration dates cannot be produced from the prompt.

What clinical and regulatory risks matter most for ATRA products in APL?

For APL ATRA regimens, the highest clinical risks are generally class-level:

• Retinoic acid syndrome: requires prompt recognition and management
• Differentiation syndrome-like toxicities in the induction phase
• Hepatic enzyme elevations and other retinoid-related AEs
• Drug-drug interactions and supportive care requirements

Regulatory risk is typically less about novel mechanism and more about:

• Product quality attributes
• Bioequivalence and consistent exposure
• Labeling compliance on dosing schedules and contraindications

Market projection: what will Vesanoid revenue do over the next 5 years?

A credible market projection requires:

• Current revenue base by geography and strength
• Share by channel (hospital procurement vs specialty pharmacy)
• Generic penetration trajectory
• Expected competitor actions (price and supply changes)
• Any ongoing regulatory or formulation changes tied to the brand

The prompt does not provide those inputs, and generating a numerical forecast without them would not meet a “hard data” standard.

Key Takeaways

• Vesanoid is ATRA (tretinoin) used in acute promyelocytic leukemia (APL), with demand driven by APL treatment patterns and generic competitive pressure.
• The prompt does not include enough verifiable, product-linked information to deliver a complete clinical trials update or a defensible 5-year market projection for the brand.
• Competitive risk is primarily generic substitution of oral tretinoin, not biosimilars.

FAQs

1. Is Vesanoid used in APL maintenance therapy or only induction?
2. How does ATRA dosing differ across APL risk groups?
3. Do generic tretinoin capsules have the same tolerability as Vesanoid?
4. What endpoints do APL trials prioritize when evaluating ATRA-based regimens?
5. What are the main safety management steps for retinoic acid syndrome in patients on ATRA?

References

1. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. U.S. Food and Drug Administration. (Accessed 2024).
2. National Cancer Institute. Acute Myeloid Leukemia Treatment (PDQ) and APL-related guidance. National Institutes of Health. (Accessed 2024).