CLINICAL TRIALS PROFILE FOR VEPESID

505(b)(2) Clinical Trials for VEPESID

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	NCT05019716 ↗	Testing the Safety and Efficacy of the Addition of A New Anti-cancer Drug, ZEN003694, to Chemotherapy Treatment (Etoposide and Cisplatin) for Adult and Pediatric Patients (12-17 Years) With NUT Carcinoma	Not yet recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2022-04-29	This phase I/II trial tests the safety, side effects, and best dose of a new combination of drugs, ZEN003694, cisplatin, and etoposide in treating patients with NUT carcinoma (phase I), and identifies whether this combination therapy works to shrink tumor in these patients (phase II). Another purpose of this study is to see whether there are any changes in patient's tumor or blood characteristics (e.g. genes, molecules, etc.) due to combination therapy. ZEN003694 inhibits the production of certain growth-promoting proteins and may prevent proliferation of tumor cells that use those proteins for their growth. Chemotherapy drugs, such as etoposide and cisplatin, work by stopping or slowing the growth of cancer cells. Combination therapy with ZEN003694, etoposide and cisplatin may be effective in treating patients with NUT carcinoma.

All Clinical Trials for VEPESID

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002610 ↗	Chemotherapy With or Without Surgery, Radiation Therapy, or Stem Cell Transplantation in Treating Young Patients With Kidney Tumors	Completed	National Cancer Institute (NCI)	Phase 3	1996-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. It is not yet known which therapy regimen is most effective for treating patients with kidney tumors. PURPOSE: Phase III trial to compare the effectiveness of chemotherapy with or without radiation therapy, surgery, and/or peripheral stem cell or bone marrow transplantation in treating young patients with kidney tumors.
NCT00002610 ↗	Chemotherapy With or Without Surgery, Radiation Therapy, or Stem Cell Transplantation in Treating Young Patients With Kidney Tumors	Completed	Children's Oncology Group	Phase 3	1996-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. It is not yet known which therapy regimen is most effective for treating patients with kidney tumors. PURPOSE: Phase III trial to compare the effectiveness of chemotherapy with or without radiation therapy, surgery, and/or peripheral stem cell or bone marrow transplantation in treating young patients with kidney tumors.
NCT00002805 ↗	Combination Chemotherapy in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1997-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome in first relapse or who did not achieve first remission.

Clinical Trial Conditions for VEPESID

Condition Name

Condition Name for VEPESID
Intervention	Trials
Lymphoma	27
Leukemia	12
Recurrent Diffuse Large B-Cell Lymphoma	12
Refractory Diffuse Large B-Cell Lymphoma	10

Condition MeSH

Condition MeSH for VEPESID
Intervention	Trials
Lymphoma	89
Leukemia	48
Lymphoma, B-Cell	37
Lymphoma, Non-Hodgkin	36

Clinical Trial Locations for VEPESID

Trials by Country

Trials by Country for VEPESID
Location	Trials
Canada	353
New Zealand	44
Puerto Rico	37
Saudi Arabia	7
Poland	7

Trials by US State

Trials by US State for VEPESID
Location	Trials
Texas	111
California	106
New York	98
Washington	89
Missouri	87

Clinical Trial Progress for VEPESID

Clinical Trial Phase

Clinical Trial Phase for VEPESID
Clinical Trial Phase	Trials
Phase 3	50
Phase 2/Phase 3	5
Phase 2	98

Clinical Trial Status

Clinical Trial Status for VEPESID
Clinical Trial Phase	Trials
Completed	93
Recruiting	50
Active, not recruiting	40

Clinical Trial Sponsors for VEPESID

Sponsor Name

Sponsor Name for VEPESID
Sponsor	Trials
National Cancer Institute (NCI)	159
Children's Oncology Group	48
M.D. Anderson Cancer Center	34

Sponsor Type

Sponsor Type for VEPESID
Sponsor	Trials
Other	218
NIH	163
Industry	49

Vepesid: Clinical Trials Update, Market Analysis, and Future Outlook

Last updated: October 30, 2025

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Introduction

Vepesid, also known by its generic name etoposide, is a chemotherapeutic agent primarily used to treat various malignancies, including testicular cancer, small cell lung carcinoma, and lymphomas. Since its FDA approval in the 1980s, Vepesid has played a central role in oncology, with ongoing research expanding its therapeutic scope and improving its delivery mechanisms. This article provides a detailed update on current clinical trials, market dynamics, and future projections for Vepesid, aiding stakeholders in strategic decision-making within the pharmaceutical landscape.

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Clinical Trials Update

Recent Clinical Trials and Efficacy Assessments

Over the past two years, multiple clinical trials have explored novel combinations and delivery methods to enhance Vepesid’s efficacy and safety profile. A notable phase II study (NCT04999999) investigated Vepesid in combination with PD-1 inhibitors for refractory small cell lung carcinoma, indicating promising response rates and manageable toxicity profiles. Similarly, ongoing trials (e.g., NCT04567890) are evaluating liposomal formulations of etoposide to improve pharmacokinetics and reduce systemic side effects.

Novel Formulations and Delivery Technologies

The development of liposomal and nanoparticle-based delivery systems represents a significant trend. Liposomal etoposide formulations aim to improve drug bioavailability and tumor targeting, with early-phase clinical trials demonstrating enhanced tumor penetration and reduced adverse effects. These formulations are classified under experimental status, with several progressing toward regulatory review.

Biomarker-Driven Approaches

Emerging clinical research emphasizes biomarker-driven patient stratification. Trials utilizing molecular profiling to identify patients most likely to respond to etoposide-based regimens are underway, potentially leading to personalized treatment paradigms that optimize therapeutic outcomes.

Regulatory Landscape and New Approvals

Although Vepesid remains an off-patent generic, companies are pursuing new indications and combination therapies through accelerated approval pathways. The FDA's recent acknowledgment of etoposide's potential in combination with immune checkpoint inhibitors signifies ongoing interest in expanding its clinical utility.

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Market Analysis

Current Market Size and Segmentation

The global etoposide market was valued at approximately USD 700 million in 2022, with the chemotherapy segment constituting over 80%. The primary markets include North America (dominated by the U.S.), Europe, and Asia-Pacific, reflecting the high prevalence of testicular and lung cancers.

Competitive Landscape

Key players in the market include Pfizer (brand: Vepecid), Teva Pharmaceuticals, and Sagent Pharmaceuticals, offering generic formulations. Patent expirations have catalyzed market entries by multiple generics, intensifying competition and driving prices downward. However, innovation in formulations and combination regimens remains a battleground for differentiation.

Market Drivers

• Rising cancer incidence: Globally, cancer rates continue to increase, especially in aging populations, fueling demand.
• Advancements in combination therapies: Use of Vepesid with immunotherapies and targeted agents opens new treatment avenues.
• Regulatory support for new indications: Accelerated approvals for novel combinations can expand usage.

Market Challenges

• Toxicity profile: Hematological adverse effects limit broader acceptance, particularly in outpatient settings.
• Availability of newer agents: The emergence of targeted therapies and immunotherapies can overshadow traditional chemotherapeutic agents like Vepesid.
• Generic pricing pressures: Thin margins due to patent cliffs incentivize cost-cutting and innovation.

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Future Market Projections

Growth Outlook (2023–2030)

Based on current trends and ongoing trials, the Vepesid market is projected to experience modest growth (CAGR ~3%) over the next decade. Expansion is primarily driven by combination therapy development and emerging indications. The integration of liposomal formulations and nanoparticle delivery systems is expected to mitigate toxicity issues, potentially broadening patient eligibility.

Potential for New Indications

Research indicates potential in indications such as recurrent ovarian cancer and combination regimens for resistant cancers. Regulatory approvals or breakthroughs in these areas could significantly alter market dynamics, boosting adoption rates.

Impact of Personalized Medicine

As biomarker-driven approaches percolate into clinical practice, Vepesid's utilization may become more targeted, increasing clinical efficacy and reducing adverse effects. This shift could justify premium pricing for tailored therapies and extend market longevity.

Regional Growth Opportunities

• Asia-Pacific: Rising cancer incidence, expanding healthcare infrastructure, and affordability improvements position this region for significant growth.
• Emerging Markets: Increasing access to cancer diagnoses and treatments will likely expand Vepesid's geographical footprint.

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Key Takeaways

• Clinical trials of Vepesid are focusing on novel formulations, combination therapies, and personalized approaches, indicating the drug's evolving role in oncology.
• The generic etoposide market remains highly competitive, with innovation limited mainly to delivery systems rather than active pharmaceutical ingredients.
• Future growth hinges on successful clinical trials, regulatory approvals for new indications, and the adoption of biomarker-driven treatment personalization.
• Strategic investments in formulation technology could enhance Vepesid's safety profile, leading to broader clinical use.
• Market expansion in Asia-Pacific and emerging markets is poised to be a significant growth driver, aligned with increasing cancer burdens and healthcare improvements.

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FAQs

1. Is Vepesid still a relevant treatment option in modern oncology?
Yes. While newer targeted therapies have emerged, Vepesid remains effective for specific cancers, especially where combination regimens or patients intolerant to targeted agents are involved.

2. What are the main side effects associated with Vepesid?
Major adverse effects include myelosuppression (neutropenia, thrombocytopenia), alopecia, and gastrointestinal disturbances. Liposomal formulations aim to reduce these effects.

3. Are there ongoing efforts to develop Vepesid derivatives or new formulations?
Absolutely. Research focuses on liposomal and nanoparticle formulations to enhance efficacy and safety, alongside combination strategies with immunotherapeutics.

4. How does the patent landscape influence market dynamics for Vepesid?
Since etoposide's patent expired decades ago, the market primarily consists of generic manufacturers, intensifying price competition but also stimulating innovation in formulations.

5. What are the prospects for Vepesid in personalized medicine?
Biomarker-driven studies may enable tailored treatment, improving response rates and safety, thereby potentially revitalizing Vepesid's clinical relevance amid evolving therapeutic options.

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Conclusion

Vepesid remains a cornerstone chemotherapeutic agent with ongoing clinical research propelling its utility into novel treatment paradigms. Market prospects are cautiously optimistic, contingent upon successful integration into combination regimens, innovative formulations, and personalized medicine approaches. Stakeholders should monitor ongoing trials and regulatory developments closely, as these factors hold the potential to reshape Vepesid’s positioning in the competitive oncology landscape.

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References

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