CLINICAL TRIALS PROFILE FOR AFINITOR

505(b)(2) Clinical Trials for AFINITOR

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	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 AFINITOR

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.
NCT00303732 ↗	Vatalanib and Everolimus in Treating Patients With Advanced Solid Tumors	Completed	Novartis	Phase 1	2004-12-01	RATIONALE: Vatalanib and everolimus may stop the growth of tumor cells by blocking blood flow to the tumor and by blocking some of the enzymes needed for cell growth. PURPOSE: This phase I trial is studying the side effects and best dose of vatalanib and everolimus and to see how well they work in treating patients with advanced solid tumors.
NCT00303732 ↗	Vatalanib and Everolimus in Treating Patients With Advanced Solid Tumors	Completed	Daniel George, MD	Phase 1	2004-12-01	RATIONALE: Vatalanib and everolimus may stop the growth of tumor cells by blocking blood flow to the tumor and by blocking some of the enzymes needed for cell growth. PURPOSE: This phase I trial is studying the side effects and best dose of vatalanib and everolimus and to see how well they work in treating patients with advanced solid tumors.
NCT00331409 ↗	Everolimus and Imatinib Mesylate in Treating Patients With Metastatic or Unresectable Kidney Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2006-01-01	RATIONALE: Everolimus and imatinib mesylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Everolimus may also block blood flow to the tumor. Giving everolimus together with imatinib mesylate may kill more tumor cells. PURPOSE: This phase II trial is studying how well giving everolimus together with imatinib mesylate works in treating patients with metastatic or unresectable kidney cancer.

Clinical Trial Conditions for AFINITOR

Condition Name

Condition Name for AFINITOR
Intervention	Trials
Breast Cancer	15
Metastatic Breast Cancer	9
Neuroendocrine Tumors	8
Metastatic Renal Cell Carcinoma	6

Condition MeSH

Condition MeSH for AFINITOR
Intervention	Trials
Carcinoma	42
Breast Neoplasms	33
Carcinoma, Renal Cell	31
Neoplasms	27

Clinical Trial Locations for AFINITOR

Trials by Country

Trials by Country for AFINITOR
Location	Trials
United States	635
Italy	74
France	46
Spain	37
Canada	35

Trials by US State

Trials by US State for AFINITOR
Location	Trials
Texas	60
California	34
Pennsylvania	29
New York	25
Florida	24

Clinical Trial Progress for AFINITOR

Clinical Trial Phase

Clinical Trial Phase for AFINITOR
Clinical Trial Phase	Trials
Phase 4	9
Phase 3	14
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for AFINITOR
Clinical Trial Phase	Trials
Completed	90
Terminated	27
Active, not recruiting	25

Clinical Trial Sponsors for AFINITOR

Sponsor Name

Sponsor Name for AFINITOR
Sponsor	Trials
Novartis	48
National Cancer Institute (NCI)	37
Novartis Pharmaceuticals	37

Sponsor Type

Sponsor Type for AFINITOR
Sponsor	Trials
Other	203
Industry	121
NIH	45

Clinical Trials Update, Market Analysis, and Projection for Afinitor (Everolimus)

Last updated: October 28, 2025

Introduction

Afinitor (generic: everolimus) is an oral mTOR inhibitor developed by Novartis, primarily approved for treating various cancers and certain non-malignant conditions. Its broad therapeutic spectrum includes advanced renal cell carcinoma (RCC), neuroendocrine tumors (NET), breast cancer, and tuberous sclerosis complex (TSC). As a cornerstone in targeted oncology, understanding its current clinical pipeline, market dynamics, and future projections is crucial for stakeholders spanning investors, healthcare providers, and competitors.

Clinical Trials Landscape: Recent Developments and Pipeline Insights

Ongoing and Recent Clinical Trials

Novartis continues to explore Afinitor’s potential across oncology and non-oncology indications through an active clinical pipeline. Recent updates include:

1. Expanded Indication Trials in Breast Cancer
   The phase III BOLERO-2 trial previously demonstrated efficacy in hormone receptor-positive, HER2-negative advanced breast cancer, leading to expanded approval. Currently, novel combination strategies, such as pairing with immune checkpoint inhibitors, are under evaluation in phase I and II trials to address resistance mechanisms and improve outcomes (NCT04554035).

2. Neuroendocrine Tumors and Pancreatic NETs
   Phase II studies are assessing the efficacy of everolimus in combination with other agents for pancreatic NETs and other gastrointestinal neuroendocrine tumors, aiming to improve progression-free survival (PFS).

3. Renal Cell Carcinoma (RCC) and Other Solid Tumors
   Several phase II and III trials involve Afinitor in advanced RCC, often combined with tyrosine kinase inhibitors or immune therapies, reflecting ongoing interest in enhancing response durability.

4. Rare Genetic Disorders and Tuberous Sclerosis Complex (TSC)
   The EXIST-1 trial established everolimus’s role in treating TSC-associated subependymal giant cell astrocytomas (SEGA). Current phase III studies are evaluating its efficacy in other TSC manifestations, such as renal angiomyolipomas.

Emerging Clinical Data and Results

• Efficacy Trends: Recent preliminary data point to improved PFS in combination therapies, with manageable safety profiles, though definitive results are pending from larger studies.
• Safety Profile: The adverse effects primarily involve stomatitis, infections, fatigue, and non-infectious pneumonitis, consistent with prior findings, which continue to inform dosing and patient selection.

Regulatory and Approval Status

While Afinitor maintains approval for RCC, breast cancer, and TSC-related indications, ongoing clinical data may support additional label expansions in the coming years. Regulatory reviews are underway for combination regimens targeting resistant or previously untreated populations.

Market Analysis of Afinitor

Market Size and Dynamics

The global oncology drug market is estimated to reach approximately $197 billion by 2025, with targeted therapies accounting for a significant share due to their efficacy and specificity [1]. Afinitor commands a robust market position within mTOR inhibitors, which include everolimus, temsirolimus, and others.

Key market segments include:

• Renal Cell Carcinoma (RCC): The RCC market was valued at over $2.3 billion in 2021, with Afinitor representing a significant portion due to its early approval and established efficacy.
• Neuroendocrine Tumors: The NET segment is smaller but rapidly growing, driven by increased diagnosis and approval of targeted therapies.
• Breast Cancer: As part of the broader hormonal and targeted therapy framework, Afinitor’s contribution is substantial, particularly for hormone receptor-positive subtypes.

Market Drivers

• Growing Incidence of Targeted Cancers: Increasing rates of RCC, breast cancer, and NETs promote higher demand.
• Expanded Indications and Label Extensions: Ongoing clinical trials could lead to new approvals, broadening market reach.
• Combination Therapy Approaches: Synergistic regimens could enhance efficacy and market penetration.

Market Challenges

• Generic Competition: Following patent expiration, generic formulations may erode Afinitor’s market share.
• Side Effect Management: Toxicity profile necessitates careful patient selection and management, potentially limiting usage.
• Pricing and Healthcare Policies: Cost considerations in healthcare systems influence reimbursement and adoption rates.

Competitive Landscape

Afinitor’s primary competitors include other mTOR inhibitors like temsirolimus (Torisel), ridaforolimus, and emerging agents targeting similar pathways. Immunotherapy agents such as immune checkpoint inhibitors are increasingly integrated into treatment regimens, posing competitive pressures.

Market Projection: Future Outlook for Afinitor

Forecast Assumptions

• The global oncology market will sustain a compound annual growth rate (CAGR) of approximately 7% through 2030.
• Clinical trial outcomes favoring combination therapies and novel indications will support label expansions.
• Patent expiry periods are anticipated around 2024-2026, with subsequent generic entries influencing sales.

Projected Revenue Trajectory

• 2023-2025: A moderate growth trajectory, supported by existing indications and potential label expansions, with sales estimated at $1.5 billion to $2.0 billion annually.
• 2026-2030: Post-patent expiration, revenues may decline by 15-25% annually due to generic competition unless offset by new indications or formulations.
• Long-Term Outlook: Incorporation of Afinitor into combination regimens for resistant cancers may stabilize or renew growth, especially if new approvals occur.

Strategic Considerations

Novartis is investing in clinical trials for combinations with immune checkpoint inhibitors and other targeted agents, aiming to extend Afinitor’s lifecycle and market relevance. Partnerships and licensing agreements may further augment its reach and revenue streams.

Key Takeaways

• Robust Clinical Pipeline: Afinitor’s ongoing trials in breast, RCC, and neuroendocrine tumors could facilitate indication expansions, maintaining its relevance.
• Market Position: Currently a leading mTOR inhibitor in oncology, though facing impending generic challenges.
• Growth Drivers: Rising cancer incidences, innovative combination strategies, and potential new approvals underpin future growth.
• Market Risks: Patent expiry, competition from immunotherapies, and adverse event management remain critical considerations.
• Strategic Focus: Leveraging clinical data to demonstrate benefits of combination therapies and exploring new indications will be vital for sustained success.

FAQs

1. What are the primary indications for Afinitor?
Afinitor is approved for advanced renal cell carcinoma, unresectable or metastatic neuroendocrine tumors of pancreatic origin, certain breast cancers, and TSC-associated SEGA.

2. Are there ongoing trials exploring Afinitor in new cancer indications?
Yes. Current trials investigate combinations with immune checkpoint inhibitors for various solid tumors, as well as potential benefits in other rare genetic disorders associated with TSC.

3. How does Afinitor’s market share compare to other mTOR inhibitors?
Afinitor remains a market leader among mTOR inhibitors owing to its earlier approval and broad indications, though competition from agents like temsirolimus persists.

4. What is the outlook for Afinitor’s sales post-patent expiry?
Sales are expected to decline due to generic competition, unless new indications, formulations, or combination regimens significantly boost its use.

5. What are the safety considerations for Afinitor therapy?
Common adverse effects include stomatitis, infections, fatigue, and pneumonitis. Careful patient management and monitoring are essential to mitigate risks.

References

[1] Market Research Future. "Global Oncology Drugs Market." 2022.