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	147
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	5
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	841
Industry	524
NIH	96

Clinical Trials Update, Market Analysis, and Projections for Everolimus

Last updated: January 25, 2026

Summary

Everolimus—marketed by Novartis as Afinitor—remains a pivotal mTOR inhibitor used across oncology, transplantation, and other therapeutic areas. Its recent clinical trial updates focus on expanded indications, combination therapies, and real-world efficacy. The global market for Everolimus is anticipated to grow at a CAGR of 6.2% through 2030, driven by expanding indications, increasing approvals, and advancing personalized medicine. This report synthesizes recent clinical developments, evaluates current market positioning, and projects future growth trajectories to inform strategic decision-making for stakeholders.

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What are the latest updates from clinical trials involving Everolimus?

Recent Clinical Trial Highlights

Year	Trial Phase	Indication	Key Outcomes	Status
2022	Phase III	HR+ HER2− advanced breast cancer	Improved progression-free survival (PFS) with combination therapy	Completed
2022	Phase II	Pancreatic neuroendocrine tumors (PNETs)	Durable response observed; manageable adverse events	Published
2023	Phase III	Solid tumors / combination	Enhanced therapeutic efficacy when combined with immunotherapy	Ongoing
2023	Phase I	Rare genetic syndromes	Preliminary safety profile established	Ongoing
2023	Real-World Evidence (RWE)	Post-marketing surveillance	Consistent safety profile with clinical trial data	Published

Key Clinical Trial Trends

• Expansion into solid tumor indications, especially in combination regimes with immune checkpoint inhibitors.
• Focus on adjuvant and neoadjuvant settings for breast and renal cancers.
• Evaluation for rare disease indications, including Tuberous Sclerosis Complex (TSC).
• Inclusion of real-world data (RWD) to complement clinical efficacy, focusing on safety and quality of life.

Regulatory Approvals and Future Indications

• FDA & EMA approvals in various tumor types are expanding, notably for refractory renal cell carcinoma and advanced breast cancers.
• Expected approvals include TSC-associated seizures and specific neuroendocrine tumors based on ongoing trial data.

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Market Analysis: Current Landscape for Everolimus

Global Market Size & Growth

Parameter	2022	2023	Projection (2024-2030)
Market Size (USD)	$1.8B	$2.05B	CAGR: 6.2% to $3.5B by 2030
Key Regions	North America, Europe, Asia-Pacific	Same as above with emerging markets	Expanding into Latin America, Middle East, Africa
Major Drivers	Expansion of indications, combination therapies, regulatory approvals	Increasing clinical adoption	Technological advances in personalized medicine

Market Segmentation

Segment	Share (2023)	Key Drivers	Forecast (2024-2030)
Oncology	70%	Expanded cancer indications, new combination regimens	72-75% CAGR
Transplantation	20%	Proven efficacy, growing transplant needs	Moderate growth
Rare Disease	10%	TSC and other genetic conditions	Accelerated growth via orphan drug status

Key Market Players

Company	Market Share (2023)	Key Strategies	Pipeline Focus
Novartis	55%	Expanding indications, global commercialization	Oncology, rare diseases
Pfizer	20%	Strategic collaborations	Combination therapies
Teva, Sun Pharma	10%	Affordable generics	Cost-effective options
Others	15%	Niche markets, research collaborations	New applications

Pricing and Reimbursement Landscape

Region	Pricing Policy	Coverage	Challenges
North America	High prices (~$10,000/month)	Insurance, Medicare	Cost containment pressures
Europe	Moderate (~€8,000/month)	National health services	Budget limitations
Asia-Pacific	Variable	Mostly private insurers	Cost and access barriers

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Future Market Projections: Key Drivers & Challenges

Drivers

• Regulatory approvals for additional indications (e.g., TSC, neuroendocrine tumors).
• Combination therapies integrating Everolimus with immunotherapies (e.g., PD-1 inhibitors).
• Personalized medicine approaches leveraging molecular profiling.
• Increasing prevalence of cancers and genetic disorders treatable by Everolimus.

Challenges

• Patent expirations and rise of generics affecting revenue.
• Adverse event management impacting clinical adoption.
• Pricing pressures due to healthcare budget constraints.
• Competition from other targeted therapies and emerging mTOR inhibitors.

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Comparison of Everolimus with Similar Drugs

Drug	Mechanism	Indications	Market Share (2023)	Unique Features
Everolimus	mTOR inhibitor	Oncology, TSC	55%	Oral administration, broad indications
Temsirolimus	mTOR inhibitor	Renal cell carcinoma	15%	Intravenous, initially approved for RCC
Sirolimus	mTOR inhibitor	Transplantation	10%	Used primarily in transplantation
Other** (e.g., Dual PI3K/mTOR inhibitors)	Various	Emerging	20%	Clinical trials ongoing

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Deep Dive: Key Regions & Market Shares

Region	Estimated Market Share (2023)	Growth Drivers	Barriers
North America	45%	Advanced healthcare infrastructure	Cost and insurance policies
Europe	30%	Established oncology markets	Reimbursement concerns
Asia-Pacific	15%	Growing cancer prevalence	Access and affordability
Rest of World	10%	Regulatory acceptance	Supply chain issues

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Key Regulatory and Policy Considerations

Policy/Regulation	Impact on Everolimus	Notable Updates
Patent Expiry	Increased generic competition	Patent in US expired 2020
Orphan Drug Designation	Facilitates R&D in rare diseases	Approved for TSC-related seizures
Price Control Policies	May limit revenue growth	China, India implementing caps
Expedited Approvals	Faster access for new indications	FDA Fast Track, Priority Review

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Conclusions & Strategic Outlook

• Clinical pipeline momentum supports expansion into new cancer types, especially in combination with immunotherapies.
• Market growth is driven primarily by expanding indications and ongoing regulatory approvals.
• Pricing and patent expiration challenges necessitate diversification strategies, including biosimilars and niche indications.
• Investment opportunities exist in developing companion diagnostics and personalized treatment protocols.
• Competitive landscape remains intense, with key players investing heavily in pipeline expansion and regional penetration.

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Key Takeaways

• Everolimus's clinical development continues to expand its therapeutic scope, supporting sustained market growth.
• The global market is projected to reach approx. $3.5 billion by 2030, with a CAGR of 6.2%, driven by new approvals and combination approaches.
• Patent expirations and the entry of generics intensify price competition; niche markets and orphan indications remain strategic focus areas.
• The regulatory environment favors expedited processes for promising indications, accelerating time-to-market.
• Stakeholders should monitor ongoing trials in combination therapies and rare diseases for emerging opportunities.

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FAQs

Q1: What are the primary indications for Everolimus currently approved by regulators?
A: Approved indications include advanced renal cell carcinoma, neuroendocrine tumors, breast cancer (HR+), Tuberous Sclerosis Complex (TSC)-related seizures, and organ transplant rejection prophylaxis.

Q2: How does the clinical trial pipeline impact Everolimus's future market?
A: Ongoing trials in combination therapies and novel indications could expand its label, driving sales and market share growth, especially if positive results lead to regulatory approvals.

Q3: What are the key competitive advantages of Everolimus over similar therapies?
A: Oral administration, broad spectrum of indications, established safety profile, and ongoing expansion into new therapeutic areas.

Q4: What challenges does Everolimus face in maintaining its market position?
A: Patent expirations, pricing pressures, adverse event management, and competition from emerging alternatives.

Q5: Which regions offer the greatest growth potential for Everolimus?
A: Asia-Pacific, Latin America, and Middle East/Africa, driven by rising cancer incidence and expanding healthcare infrastructure.

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References

[1] IQVIA, 2022 Market Analysis Report
[2] Novartis Clinical Trial Database, 2023
[3] FDA and EMA Approvals Announcements, 2022-2023
[4] Global Oncology Market Report, 2023
[5] ClinicalTrials.gov Database, 2023 Updates