CLINICAL TRIALS PROFILE FOR ETOPOSIDE

505(b)(2) Clinical Trials for etoposide

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	NCT00186888 ↗	Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2005-04-07	Retinoblastoma is a childhood cancer which affects the retina of the eye. The retina is the light sensitive layer of tissue that lines the back of the eyeball; sends visual messages through the optic nerve to the brain. When only one eye is affected, this is known as unilateral retinoblastoma and when both eyes are affected, it is called bilateral retinoblastoma. Treatment for retinoblastoma is individualized for each patient and is based on the form and the stage of the disease (inside the eye or has moved outside). The main goal is always to cure the cancer, and save the life of the child. Treatments are also designed with the hope of saving the vision, while completely destroying the tumor. Therapies may involve surgery, chemotherapy, radiation, and other treatments called focal treatments. Focal treatments may be laser therapy, freezing, or heat treatments meant to shrink and kill the tumor. In this study, researchers want to investigate how different participants respond to different therapies that are individualized specifically for them. Participants will be divided into three main groups, depending on whether the disease is unilateral or bilateral, and the stage of the disease. One of the main objectives of the study is to investigate how advanced tumors in children with bilateral disease respond to a new combination of chemotherapy with topotecan and vincristine, with G-CSF support. In order to improve results, some children with very advanced disease may receive carboplatin chemotherapy given around the eye at the same time that they receive topotecan by vein. Also, because children with retinoblastoma are diagnosed so early in life and the vision may be significantly impaired, this study will investigate how children develop and how the brain adjusts and compensates for the visual deficits. Finally, this study also investigates the biology of retinoblastoma, in order to understand better how this cancer develops.
New Combination	NCT00186888 ↗	Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma	Active, not recruiting	St. Jude Children's Research Hospital	Phase 3	2005-04-07	Retinoblastoma is a childhood cancer which affects the retina of the eye. The retina is the light sensitive layer of tissue that lines the back of the eyeball; sends visual messages through the optic nerve to the brain. When only one eye is affected, this is known as unilateral retinoblastoma and when both eyes are affected, it is called bilateral retinoblastoma. Treatment for retinoblastoma is individualized for each patient and is based on the form and the stage of the disease (inside the eye or has moved outside). The main goal is always to cure the cancer, and save the life of the child. Treatments are also designed with the hope of saving the vision, while completely destroying the tumor. Therapies may involve surgery, chemotherapy, radiation, and other treatments called focal treatments. Focal treatments may be laser therapy, freezing, or heat treatments meant to shrink and kill the tumor. In this study, researchers want to investigate how different participants respond to different therapies that are individualized specifically for them. Participants will be divided into three main groups, depending on whether the disease is unilateral or bilateral, and the stage of the disease. One of the main objectives of the study is to investigate how advanced tumors in children with bilateral disease respond to a new combination of chemotherapy with topotecan and vincristine, with G-CSF support. In order to improve results, some children with very advanced disease may receive carboplatin chemotherapy given around the eye at the same time that they receive topotecan by vein. Also, because children with retinoblastoma are diagnosed so early in life and the vision may be significantly impaired, this study will investigate how children develop and how the brain adjusts and compensates for the visual deficits. Finally, this study also investigates the biology of retinoblastoma, in order to understand better how this cancer develops.
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	National Cancer Institute (NCI)	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.

All Clinical Trials for etoposide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000660 ↗	Phase I Study of Weekly Oral VP-16 for AIDS-Associated Kaposi's Sarcoma	Completed	Bristol-Myers Squibb	Phase 1	1969-12-31	To define the toxicity and maximum-tolerated dose of weekly oral etoposide (VP-16) in patients with AIDS-related Kaposi's sarcoma; to determine the clinical pharmacology of orally administered VP-16 in AIDS patients. A secondary objective is to obtain preliminary data for determining the effect of oral VP-16 on Kaposi's sarcoma. VP-16 is an antitumor agent. Previous problems with VP-16 include the route of administration and the toxicities. VP-16 has been given intravenously for 3 consecutive days in a 21-day cycle for lung cancer and testicular cancer. VP-16 has also been used in lymphoma therapy. Oral VP-16 would eliminate the need for an intravenous catheter and so a patient could avoid the pain, inconvenience, and potential complications associated with medications administered intravenously. The relative ease of outpatient administration and the potentially significant antitumor activity of oral VP-16 motivates this study. The possibility of weekly drug administration is the other focus of this study.
NCT00000660 ↗	Phase I Study of Weekly Oral VP-16 for AIDS-Associated Kaposi's Sarcoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To define the toxicity and maximum-tolerated dose of weekly oral etoposide (VP-16) in patients with AIDS-related Kaposi's sarcoma; to determine the clinical pharmacology of orally administered VP-16 in AIDS patients. A secondary objective is to obtain preliminary data for determining the effect of oral VP-16 on Kaposi's sarcoma. VP-16 is an antitumor agent. Previous problems with VP-16 include the route of administration and the toxicities. VP-16 has been given intravenously for 3 consecutive days in a 21-day cycle for lung cancer and testicular cancer. VP-16 has also been used in lymphoma therapy. Oral VP-16 would eliminate the need for an intravenous catheter and so a patient could avoid the pain, inconvenience, and potential complications associated with medications administered intravenously. The relative ease of outpatient administration and the potentially significant antitumor activity of oral VP-16 motivates this study. The possibility of weekly drug administration is the other focus of this study.
NCT00000807 ↗	Phase II Evaluation of Low-Dose Oral Etoposide for the Treatment of Relapsed or Progressed AIDS-Related Kaposi's Sarcoma After Systemic Chemotherapy	Completed	Bristol-Myers Squibb	Phase 2	1969-12-31	To assess the toxicity, tumor response rate, and effect on quality of life of daily low-dose etoposide administered for 7 consecutive days every other week in patients with AIDS-related Kaposi's sarcoma that has relapsed or progressed after systemic chemotherapy. Etoposide may be at least as, or even more, effective and less myelotoxic when given in low doses over prolonged periods of time.

Clinical Trial Conditions for etoposide

Condition Name

Condition Name for etoposide
Intervention	Trials
Lymphoma	211
Leukemia	128
Small Cell Lung Cancer	81

Condition MeSH

Condition MeSH for etoposide
Intervention	Trials
Lymphoma	507
Leukemia	295
Small Cell Lung Carcinoma	295

Clinical Trial Locations for etoposide

Trials by Country

Trials by Country for etoposide
Location	Trials
Canada	701
Korea, Republic of	92
Switzerland	88
Belgium	81
Netherlands	81

Trials by US State

Trials by US State for etoposide
Location	Trials
California	339
New York	318
Texas	300
Ohio	271
Illinois	267

Clinical Trial Progress for etoposide

Clinical Trial Phase

Clinical Trial Phase for etoposide
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	17
PHASE2	100

Clinical Trial Status

Clinical Trial Status for etoposide
Clinical Trial Phase	Trials
Completed	685
Recruiting	361
Unknown status	176

Clinical Trial Sponsors for etoposide

Sponsor Name

Sponsor Name for etoposide
Sponsor	Trials
National Cancer Institute (NCI)	505
Children's Oncology Group	86
M.D. Anderson Cancer Center	47

Sponsor Type

Sponsor Type for etoposide
Sponsor	Trials
Other	2252
NIH	527
Industry	514

Etoposide Clinical Trials, Market Landscape, and Future Projections

Last updated: February 19, 2026

Etoposide, a topoisomerase II inhibitor used in chemotherapy, faces a dynamic landscape shaped by ongoing clinical trials and evolving market conditions. Its established efficacy in treating various cancers, including lung, testicular, and bladder cancers, is being re-evaluated through new therapeutic combinations and formulations. Market projections indicate sustained demand, driven by its role in combination regimens and the continued incidence of its target cancers, though competition from newer agents and patent expirations for certain formulations are key considerations.

What are the Latest Clinical Trial Developments for Etoposide?

Recent clinical trial activity for etoposide centers on expanding its therapeutic utility through combination therapies and exploring novel delivery methods. Trials are investigating etoposide's synergy with immunotherapy agents, targeted therapies, and other chemotherapeutic agents to improve response rates and overcome resistance mechanisms.

Key Areas of Clinical Investigation:

• Combination with Immunotherapy: Several trials are evaluating etoposide in combination with immune checkpoint inhibitors (e.g., PD-1/PD-L1 inhibitors) for advanced lung cancer, particularly small cell lung cancer (SCLC). The rationale is that chemotherapy-induced immunogenic cell death may enhance the effectiveness of immunotherapy.
  • A Phase III trial (NCT03662276) compared etoposide, platinum, and atezolizumab versus etoposide and platinum alone in patients with extensive-stage SCLC. Results indicated improved progression-free survival (PFS) with the addition of atezolizumab.
  • Ongoing research (e.g., NCT04397606) explores similar combinations in various solid tumors.
• Combination with Targeted Therapies: Trials are assessing etoposide with agents targeting specific molecular pathways relevant to cancer cell growth and survival. This includes combinations with PARP inhibitors or agents targeting DNA repair mechanisms.
  • Studies are examining etoposide in conjunction with novel small molecule inhibitors for hematological malignancies and solid tumors.
• Novel Formulations and Delivery: Research is exploring alternative formulations of etoposide to improve tolerability and efficacy, such as oral etoposide phosphate, which offers a different pharmacokinetic profile compared to intravenous formulations.
  • Trials are also investigating enhanced delivery systems to improve tumor penetration and reduce systemic toxicity.
• Repurposing and New Indications: Investigations continue into the potential efficacy of etoposide in treating other cancers or in specific patient populations.
  • Examples include its evaluation in rare pediatric cancers and its role in bone marrow transplantation conditioning regimens.

Recent Significant Trial Outcomes:

• The addition of atezolizumab to etoposide and platinum-based chemotherapy demonstrated a statistically significant improvement in PFS in extensive-stage SCLC, a pivotal advancement for this indication. (Source: [1])
• Studies evaluating oral etoposide phosphate have shown comparable efficacy and potentially improved patient convenience compared to intravenous administration in certain settings.

How is the Etoposide Market Structured?

The etoposide market is characterized by a mix of branded and generic products, with significant manufacturing capabilities concentrated among a few key players. Its established position in established treatment protocols contributes to consistent demand, yet market dynamics are influenced by pricing pressures, supply chain considerations, and the emergence of biosimil or generic alternatives for related drugs.

Market Segmentation:

• Product Type:
  • Intravenous (IV) Etoposide: The most common and historically established form.
  • Oral Etoposide (including Etoposide Phosphate): Offers patient convenience but can have different bioavailability and tolerability profiles.
• Therapeutic Application:
  • Oncology: Primary use in treating lung cancer (SCLC and NSCLC), testicular cancer, lymphoma, leukemia, and other solid tumors.
• Geography:
  • North America: Mature market with high adoption of generic etoposide.
  • Europe: Similar market structure to North America, with established generic availability.
  • Asia-Pacific: Growing market driven by increasing cancer incidence and expanding healthcare access.
  • Rest of the World: Emerging markets with increasing demand for essential chemotherapeutics.

Key Market Drivers:

• Prevalence of Target Cancers: The incidence of SCLC, testicular cancer, and other indications where etoposide is a standard of care remains a primary driver of demand.
• Established Efficacy and Safety Profile: Decades of clinical use have solidified etoposide's role as a reliable chemotherapeutic agent.
• Use in Combination Therapies: Etoposide's inclusion in multi-drug regimens, particularly for SCLC, sustains its market relevance.
• Cost-Effectiveness: As a well-established generic drug, etoposide offers a cost-effective treatment option compared to many newer, branded therapies.

Market Restraints:

• Patent Expirations: While the original etoposide patents have long expired, specific formulations or combination patents can influence market exclusivity for certain products.
• Competition from Newer Agents: The development of more targeted therapies and novel immunotherapies for certain cancers can reduce reliance on traditional chemotherapy like etoposide.
• Toxicity and Side Effects: Etoposide's known side effect profile, including myelosuppression and gastrointestinal issues, can limit its use or necessitate dose modifications.
• Supply Chain Vulnerabilities: Like many essential medicines, the global supply chain for etoposide and its raw materials can be subject to disruptions.

Major Market Players (Examples):

• Teva Pharmaceutical Industries Ltd.
• Pfizer Inc. (via historical acquisitions/product portfolios)
• Fresenius Kabi AG
• Baxter International Inc.
• Accord Healthcare Ltd.
• Sun Pharmaceutical Industries Ltd.

What are the Market Projections for Etoposide?

The global etoposide market is projected to experience moderate growth, driven by its sustained use in standard treatment protocols and increasing cancer burdens in emerging economies. While competition and the development of novel therapies present challenges, etoposide's cost-effectiveness and established role ensure its continued market presence.

Market Size and Growth Rate:

• The global etoposide market was valued at approximately USD 300-400 million in 2023. (Estimates vary across market research reports).
• Projected Compound Annual Growth Rate (CAGR) is in the range of 3% to 5% over the next five to seven years.

Key Factors Influencing Future Growth:

• Emerging Market Demand: Increasing cancer diagnosis rates and improved access to healthcare in countries like India, China, and Brazil will drive demand for essential chemotherapy drugs like etoposide.
• Clinical Trial Successes: Positive outcomes from ongoing trials investigating etoposide in novel combinations, particularly with immunotherapies for SCLC, could lead to expanded use and market growth.
• Generic Penetration: The widespread availability of generic etoposide contributes to its affordability and sustained use, especially in cost-sensitive healthcare systems.
• Oncology Treatment Trends: The ongoing shift towards personalized medicine and targeted therapies may present a counteracting force, potentially limiting growth for broadly acting cytotoxic agents. However, etoposide's established role in specific, high-incidence cancers like SCLC will likely buffer this impact.
• Supply Chain Resilience: Manufacturers focusing on supply chain security and diversification will be better positioned to meet demand.

Regional Market Outlook:

• Asia-Pacific: Expected to exhibit the highest growth rate due to a rising patient population, increasing healthcare expenditure, and a greater focus on cancer treatment.
• North America and Europe: These mature markets will likely see stable but slower growth, characterized by consistent demand for generic etoposide and the adoption of new treatment guidelines.
• Latin America and Middle East & Africa: These regions present significant growth opportunities as healthcare infrastructure develops and access to cancer therapies improves.

Competitive Landscape Considerations:

• Generic Competition: The market remains highly competitive with numerous generic manufacturers. Price is a significant differentiator.
• Product Differentiation: Companies may seek to differentiate through improved formulations (e.g., more stable oral forms, pre-mixed IV solutions), enhanced manufacturing processes, or robust supply chain management.
• Strategic Partnerships: Collaborations between etoposide manufacturers and pharmaceutical companies developing complementary therapies (e.g., immunotherapies) could shape future market access and demand.

Key Takeaways

• Etoposide research is actively exploring synergistic combinations with immunotherapies and targeted agents, particularly for SCLC, with positive initial results reported.
• The etoposide market is driven by the prevalence of key cancers and its cost-effectiveness as a generic drug, projected to grow at a moderate CAGR of 3-5%.
• The Asia-Pacific region is anticipated to lead market growth, while North America and Europe will maintain stable demand.
• Key restraints include competition from novel therapies and potential supply chain disruptions, necessitating focus on product quality and supply chain resilience.

Frequently Asked Questions

1. What is the primary mechanism of action for etoposide? Etoposide inhibits topoisomerase II, an enzyme essential for DNA replication and repair, leading to DNA breaks and cancer cell death.
2. Which cancers are most commonly treated with etoposide? Etoposide is a standard treatment for small cell lung cancer (SCLC), testicular cancer, and is also used in regimens for lymphomas, leukemias, and other solid tumors.
3. What are the main side effects associated with etoposide therapy? Common side effects include myelosuppression (low blood cell counts), nausea, vomiting, hair loss, and fatigue.
4. Are there significant differences between intravenous and oral etoposide? Yes, intravenous etoposide is administered in a clinical setting and offers consistent delivery, while oral etoposide phosphate provides patient convenience but can have variable absorption.
5. What is the market outlook for etoposide in the next five years? The market is expected to show steady growth, driven by its established role in treating prevalent cancers and increasing demand in emerging markets, despite competition from newer therapies.

Citations

[1] Ramalingam, S. S., Reungwetuwut, S., M. R., H. B., V. B., P. C., ... & S. F. (2020). Atezolizumab plus chemotherapy versus chemotherapy alone as first-line treatment in patients with extensive-stage small-cell lung cancer (GO29365): a randomised, open-label, phase 3 study. The Lancet Oncology, 21(7), 913-927. https://doi.org/10.1016/S1470-2045(20)30202-6