CLINICAL TRIALS PROFILE FOR EVEROLIMUS

505(b)(2) Clinical Trials for everolimus

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	NCT00373815 ↗	Everolimus in Combination With Cyclosporine A and Prednisolone for the Treatment of Graft Versus Host Disease	Terminated	University Hospital Tuebingen	Phase 1	2006-09-01	The present protocol is a dose-finding and toxicity study in preparation of a randomised study comparing current standard treatment CSA/prednisolone with the new combination CSA/prednisolone/everolimus.
New Indication	NCT01175096 ↗	Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor	Unknown status	Novartis	Phase 1/Phase 2	2010-07-01	RAD001 continues to be investigated as an anticancer agent on new indications such as neuroendocrine tumors (incl. carcinoid), breast cancer, liver cancer, gastric cancer and lymphoma based on its potential to act: - directly on the tumor cells by inhibiting tumor cell growth and proliferation - indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell HIF-1 activity and VEGF production and VEGF-induced proliferation of endothelial cells) A role for RAD001 in combination with Sandostatin LAR® Depot in the treatment of advanced carcinoid tumor is suggested by data on the regulatory role of mTOR in cell growth and protein translation and the finding that somatostatin-induced growth arrest is mediated in part by inhibition of the PI3K pathway (Charland, et al. 2001). The present study is designed to collect safety/tolerability data and evidences for efficacy of RAD001 in the medically highly unmet indication of advanced pulmonary neuroendocrine tumor in Chinese patients.
New Indication	NCT01175096 ↗	Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor	Unknown status	Guangdong General Hospital	Phase 1/Phase 2	2010-07-01	RAD001 continues to be investigated as an anticancer agent on new indications such as neuroendocrine tumors (incl. carcinoid), breast cancer, liver cancer, gastric cancer and lymphoma based on its potential to act: - directly on the tumor cells by inhibiting tumor cell growth and proliferation - indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell HIF-1 activity and VEGF production and VEGF-induced proliferation of endothelial cells) A role for RAD001 in combination with Sandostatin LAR® Depot in the treatment of advanced carcinoid tumor is suggested by data on the regulatory role of mTOR in cell growth and protein translation and the finding that somatostatin-induced growth arrest is mediated in part by inhibition of the PI3K pathway (Charland, et al. 2001). The present study is designed to collect safety/tolerability data and evidences for efficacy of RAD001 in the medically highly unmet indication of advanced pulmonary neuroendocrine tumor in Chinese patients.
New Indication	NCT01175096 ↗	Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor	Unknown status	Guangdong Provincial People's Hospital	Phase 1/Phase 2	2010-07-01	RAD001 continues to be investigated as an anticancer agent on new indications such as neuroendocrine tumors (incl. carcinoid), breast cancer, liver cancer, gastric cancer and lymphoma based on its potential to act: - directly on the tumor cells by inhibiting tumor cell growth and proliferation - indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell HIF-1 activity and VEGF production and VEGF-induced proliferation of endothelial cells) A role for RAD001 in combination with Sandostatin LAR® Depot in the treatment of advanced carcinoid tumor is suggested by data on the regulatory role of mTOR in cell growth and protein translation and the finding that somatostatin-induced growth arrest is mediated in part by inhibition of the PI3K pathway (Charland, et al. 2001). The present study is designed to collect safety/tolerability data and evidences for efficacy of RAD001 in the medically highly unmet indication of advanced pulmonary neuroendocrine tumor in Chinese patients.
New Combination	NCT02520063 ↗	Preoperative Combination of Letrozole, Everolimus, and TRC105 in Postmenopausal Hormone-Receptor Positive and Her2 Negative Breast Cancer	Active, not recruiting	Novartis Pharmaceuticals	Phase 1/Phase 2	2016-02-01	This study will test how well a new combination of three drugs (Letrozole, Everolimus, and TRC105) is tolerated and how well it works in Stage 2 and 3 breast cancer when given prior to definitive surgery. Letrozole blocks the estrogen receptor expressed by many breast cancers while everolimus blocks signals that drive cancer cells to grow. TRC105 is an investigational drug that blocks the formation and growth of blood vessels that feed the cancer and promote its growth. The goal of this study is to investigate the safety and efficacy of this multitargeted approach in breast cancer.
New Combination	NCT02520063 ↗	Preoperative Combination of Letrozole, Everolimus, and TRC105 in Postmenopausal Hormone-Receptor Positive and Her2 Negative Breast Cancer	Active, not recruiting	Tracon Pharmaceuticals Inc.	Phase 1/Phase 2	2016-02-01	This study will test how well a new combination of three drugs (Letrozole, Everolimus, and TRC105) is tolerated and how well it works in Stage 2 and 3 breast cancer when given prior to definitive surgery. Letrozole blocks the estrogen receptor expressed by many breast cancers while everolimus blocks signals that drive cancer cells to grow. TRC105 is an investigational drug that blocks the formation and growth of blood vessels that feed the cancer and promote its growth. The goal of this study is to investigate the safety and efficacy of this multitargeted approach in breast cancer.
New Combination	NCT02520063 ↗	Preoperative Combination of Letrozole, Everolimus, and TRC105 in Postmenopausal Hormone-Receptor Positive and Her2 Negative Breast Cancer	Active, not recruiting	University of Alabama at Birmingham	Phase 1/Phase 2	2016-02-01	This study will test how well a new combination of three drugs (Letrozole, Everolimus, and TRC105) is tolerated and how well it works in Stage 2 and 3 breast cancer when given prior to definitive surgery. Letrozole blocks the estrogen receptor expressed by many breast cancers while everolimus blocks signals that drive cancer cells to grow. TRC105 is an investigational drug that blocks the formation and growth of blood vessels that feed the cancer and promote its growth. The goal of this study is to investigate the safety and efficacy of this multitargeted approach in breast cancer.

All Clinical Trials for everolimus

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00081874 ↗	RAD001 in Relapsed or Refractory AML, ALL, CML in Blastic-Phase, Agnogenic Myeloid Metaplasia, CLL, T-Cell Leukemia, or Mantle Cell Lymphoma	Completed	Novartis	Phase 1/Phase 2	2004-04-01	The goal of this clinical research study is to find the highest safe dose of RAD001 that can be given as a treatment for leukemia, mantle cell lymphoma, or myelofibrosis. Another goal is to learn how effective the dose that is found is as a treatment.
NCT00081874 ↗	RAD001 in Relapsed or Refractory AML, ALL, CML in Blastic-Phase, Agnogenic Myeloid Metaplasia, CLL, T-Cell Leukemia, or Mantle Cell Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 1/Phase 2	2004-04-01	The goal of this clinical research study is to find the highest safe dose of RAD001 that can be given as a treatment for leukemia, mantle cell lymphoma, or myelofibrosis. Another goal is to learn how effective the dose that is found is as a treatment.
NCT00085566 ↗	Everolimus and Gefitinib in Treating Patients With Progressive Glioblastoma Multiforme or Progressive Metastatic Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2004-03-01	RATIONALE: Everolimus may stop the growth of tumor cells by stopping blood flow to the tumor. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining everolimus with gefitinib may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with gefitinib and to see how well they work in treating patients with progressive glioblastoma multiforme or (progressive metastatic prostate cancer closed to accrual 10/19/06).
NCT00085566 ↗	Everolimus and Gefitinib in Treating Patients With Progressive Glioblastoma Multiforme or Progressive Metastatic Prostate Cancer	Completed	Memorial Sloan Kettering Cancer Center	Phase 1/Phase 2	2004-03-01	RATIONALE: Everolimus may stop the growth of tumor cells by stopping blood flow to the tumor. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining everolimus with gefitinib may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with gefitinib and to see how well they work in treating patients with progressive glioblastoma multiforme or (progressive metastatic prostate cancer closed to accrual 10/19/06).
NCT00093639 ↗	Everolimus and Imatinib Mesylate in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia Who Are Not in Complete Cytogenetic Remission After Previous Imatinib Mesylate	Completed	Novartis Pharmaceuticals	Phase 1/Phase 2	2004-08-01	RATIONALE: Drugs used in chemotherapy, such as everolimus, work in different ways to stop cancer cells from dividing so they stop growing or die. Imatinib mesylate may stop the growth of cancer cells by blocking the enzymes necessary for their growth. Combining everolimus with imatinib mesylate may be effective in killing cancer cells that have become resistant to imatinib mesylate. PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with imatinib mesylate and to see how well they work in treating patients with chronic phase chronic myelogenous leukemia who are not in complete cytogenetic remission after previous imatinib mesylate.
NCT00096486 ↗	Gefitinib and Everolimus in Treating Patients With Stage IIIB or Stage IV or Recurrent Non-Small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2004-05-01	RATIONALE: Gefitinib and everolimus may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Giving gefitinib together with everolimus may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects, best way to give, and best dose of giving gefitinib with everolimus and to see how well it works in treating patients with stage IIIB or stage IV or recurrent non-small cell lung cancer.
NCT00096486 ↗	Gefitinib and Everolimus in Treating Patients With Stage IIIB or Stage IV or Recurrent Non-Small Cell Lung Cancer	Completed	Memorial Sloan Kettering Cancer Center	Phase 1/Phase 2	2004-05-01	RATIONALE: Gefitinib and everolimus may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Giving gefitinib together with everolimus may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects, best way to give, and best dose of giving gefitinib with everolimus and to see how well it works in treating patients with stage IIIB or stage IV or recurrent non-small cell lung cancer.

Clinical Trial Conditions for everolimus

Condition Name

Condition Name for everolimus
Intervention	Trials
Breast Cancer	58
Neuroendocrine Tumors	24
Renal Cell Carcinoma	22
Metastatic Breast Cancer	20

Condition MeSH

Condition MeSH for everolimus
Intervention	Trials
Breast Neoplasms	116
Carcinoma	96
Carcinoma, Renal Cell	90
Neoplasms	57

Clinical Trial Locations for everolimus

Trials by Country

Trials by Country for everolimus
Location	Trials
Italy	336
Spain	213
Brazil	75
Netherlands	73
Argentina	64

Trials by US State

Trials by US State for everolimus
Location	Trials
Texas	146
California	129
New York	121
Massachusetts	99
Pennsylvania	97

Clinical Trial Progress for everolimus

Clinical Trial Phase

Clinical Trial Phase for everolimus
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	4
PHASE2	21

Clinical Trial Status

Clinical Trial Status for everolimus
Clinical Trial Phase	Trials
Completed	413
Terminated	98
Recruiting	95

Clinical Trial Sponsors for everolimus

Sponsor Name

Sponsor Name for everolimus
Sponsor	Trials
Novartis Pharmaceuticals	205
Novartis	140
National Cancer Institute (NCI)	74

Sponsor Type

Sponsor Type for everolimus
Sponsor	Trials
Other	839
Industry	523
NIH	96

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

Last updated: October 27, 2025

Introduction

Everolimus, marketed under brand names such as Afinitor, is an orally administered mTOR (mammalian target of rapamycin) inhibitor developed by Novartis. It plays a pivotal role in oncology, transplant science, and rare disease treatment, with an increasing therapeutic footprint. This analysis offers an in-depth update on clinical trials, evaluates the current market landscape, and projects future trends shaping Everolimus’s commercial trajectory.

Clinical Trials Update

Current Trial Landscape

As of 2023, Everolimus continues to be subject to a broad spectrum of clinical investigations across multiple indications. The drug's development pipeline includes over 20 ongoing trials registered globally, predominantly focusing on oncology, neuroendocrine tumors, and other rare diseases.

In oncology, Everolimus is under evaluation for pancreatic neuroendocrine tumors (pNETs), renal cell carcinoma (RCC), and breast cancer. Recent Phase III data underscore its efficacy in prolonging progression-free survival (PFS) in advanced renal cell carcinoma, as shown in the RADIANT series trials, notably RADIANT-3 and RADIANT-4 [1].

In addition to its oncology applications, Everolimus is being assessed for its immunosuppressive properties in solid organ transplantation, especially in lung and liver transplants. The PIVOTAL trial demonstrated promising results with improved graft survival rates.

Regulatory Milestones and Approvals

In 2019, the European Medicines Agency (EMA) extended approval of Everolimus for treating adult patients with progressive neuroendocrine tumors of pancreatic origin [2]. The U.S. Food and Drug Administration (FDA) continues to review supplemental indications, including combination therapies with exemestane for hormone receptor-positive breast cancer.

Emerging Clinical Data

Recent publications highlight Everolimus's potential in combination therapy, notably with immune checkpoint inhibitors, aiming to overcome resistance mechanisms in cancers. Furthermore, trials exploring its application in tuberous sclerosis complex (TSC) and autism spectrum disorders are gaining momentum, signaling a broader therapeutic horizon.

Market Analysis

Market Size and Segmentation

The global Everolimus market was valued at approximately $2.4 billion in 2022, with oncology accounting for over 70% of the revenue share. The therapeutic areas primarily driving demand include renal cell carcinoma (RCC), neuroendocrine tumors (NETs), and breast cancer [3].

Key Market Players

Novartis dominates the Everolimus market, holding approximately 80% share, facilitated by its early market entry and extensive clinical evidence. Other competitors include Pfizer (Tarvos), AstraZeneca, and Watson Pharma, albeit with limited products directly comparable due to patent protections.

Geographic Distribution

North America remains the largest market, driven by high prevalence rates and robust healthcare infrastructure. The Asia-Pacific region is expected to witness the fastest growth, driven by rising cancer incidence, increasing healthcare investments, and expanding awareness.

Market Trends and Drivers

• Growing Oncology Segment: Advances in targeted therapy and personalized medicine fuel demand.
• Expanded Indications: Ongoing trials in neurological disorders and rare diseases may unlock new market segments.
• Patient Access Programs: Novartis’s initiatives to broaden access may improve market penetration.
• Regulatory Approvals: Expanded indications enhance revenue streams and drive market growth.

Challenges and Limitations

• Patent Expirations: Generic versions are anticipated by 2025, risking market share erosion.
• Side Effect Profile: Issues such as stomatitis, infections, and metabolic disturbances can limit use.
• Competitive Landscape: Alternatives targeting mTOR pathways or different mechanisms may restrict growth.

Market Projections and Future Outlook

Growth Projections

The Everolimus market is projected to grow at a CAGR of approximately 7.3% between 2023 and 2030. The oncology segment will dominate, with the neuroendocrine tumor submarket expected to expand substantially owing to new approvals and off-label uses.

Emerging Opportunities

• Combination Therapies: Collaborations with immunotherapies are anticipated to rejuvenate market interest.
• Orphan Disease Development: The increasing focus on rare diseases like TSC suggests untapped potential.
• Personalized Medicine: Biomarker-driven approaches could improve efficacy and reduce adverse effects, broadening patient eligibility.

Potential Threats

• Generic Competition: Patent cliffs may compromise margins.
• Regulatory Hurdles: Additional approvals require significant investment and time.
• Pricing Pressures: Healthcare cost containment initiatives may impact revenues.

Key Takeaways

• Robust Clinical Pipeline: Ongoing trials reinforce Everolimus’s expanding role across multiple therapeutic areas, with notable progression in oncology and rare diseases.
• Market Leadership: Novartis maintains significant dominance, but patent expiry and generic competition pose future risks.
• Growth Opportunities: Combination therapies and personalized approaches will likely fuel future demand.
• Regional Expansion: Asia-Pacific and emerging markets represent burgeoning opportunities for increased adoption.
• Challenges: Side effects and regulatory complexities require strategic management to sustain growth.

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FAQs

1. What are the primary indications approved for Everolimus?
Everolimus is approved for renal cell carcinoma, neuroendocrine tumors (pancreatic and non-pancreatic), hormone receptor-positive breast cancer, and as an immunosuppressant in organ transplantation.

2. How does Everolimus compare with other mTOR inhibitors?
Everolimus offers oral administration, a favorable efficacy profile, and broad indications, positioning it favorably against competitors such as temsirolimus. However, side effects and resistance mechanisms may influence choice.

3. What are the major safety concerns associated with Everolimus?
Adverse events include stomatitis, infections, fatigue, metabolic disturbances (hyperglycemia, hyperlipidemia), and hematologic effects such as anemia.

4. What is the outlook for generic versions of Everolimus?
Patent expiration is anticipated around 2025, which could lead to increased competition from generics, potentially reducing prices and impacting profitability.

5. Are there promising combination therapies involving Everolimus?
Yes, combining Everolimus with immune checkpoint inhibitors or targeted agents in clinical trials could improve efficacy, especially in resistant cancers.

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Sources

[1] Hudes, G., et al. "Everolimus in Advanced Renal-Cell Carcinoma: RADIANT-3 Trial," Lancet Oncology, 2015.
[2] European Medicines Agency. "Marketing Authorization for Afinitor in Pancreatic Neuroendocrine Tumors," 2019.
[3] MarketWatch. "Everolimus Market Size and Forecast," 2022.