CLINICAL TRIALS PROFILE FOR TYKERB

505(b)(2) Clinical Trials for TYKERB

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
OTC	NCT00496366 ↗	Capecitabine (Xeloda) and Lapatinib (Tykerb) as First-line Therapy in HER2/Neu-positive Breast Cancer	Terminated	National Cancer Institute (NCI)	Phase 2	2007-07-23	Subjects with advanced or metastatic (spread to other parts of the body) breast cancer that is HER2/neu-positive will take part in this study. This type of breast cancer has a high amount of a protein called HER2. HER2 is part of a family of receptors found on both cancer and normal cells. This family of receptors is important for cell growth and is found in many tumor types. The purpose of this research study is to compare an approved treatment for breast cancer capecitabine, also called Xeloda®, to the combination of capecitabine plus an experimental drug, lapatinib also known as Tykerb®, for treatment of advanced or metastatic breast cancer that is HER2/neu-positive.Capecitabine is an approved type of chemotherapy used to treat certain cancers including breast cancer. Capecitabine fights cancer by interfering with the ability of cells to divide and tumor growth. Lapatinib (Tykerb®) is considered "investigational", which means the drug has not been approved by the US Food and Drug Administration (FDA) for sale as a prescription or over-the-counter medication. Lapatinib may slow or stop cancer cells from growing by inhibiting the growth of cancer cells. However, this theory has not been proven. The addition of the study drug (lapatinib) to capecitabine may help stop cancer cells as well as or better than capecitabine alone. Other studies have demonstrated activity and tolerability of lapatinib either alone or in combination with capecitabine in the treatment of breast cancer.Subjects will receive capecitabine and lapatinib. A treatment period will be 21 days long. This period is known as a "cycle". All medications will be given by mouth. Subjects will take capecitabine for 2 weeks straight (Day 1-14) followed by a 1 week without capecitabine (Day 15-21). Doses of lapatinib will be taken daily continuously for 21 days (Day 1-Day 21) which means that subjects will still take lapatinib on the week that they do not take capecitabine (Day 15-21). Subjects will continue to receive these medications unless they experience severe, serious and/or excessive side effects, the cancer becomes worse, the subjects wishes to no longer participate or the study doctor feels it is not in the best interest to continue treatment.Tests and procedures such as physical exam, blood tests, CT or MRI, ECG, ECHO and/or MUGA tests will be conducted at one or more of the following time points: before the study starts, before each cycle, every 6 and 12 weeks, and after the last dose of capecitabine/lapatinib treatment.
OTC	NCT00496366 ↗	Capecitabine (Xeloda) and Lapatinib (Tykerb) as First-line Therapy in HER2/Neu-positive Breast Cancer	Terminated	Rutgers Cancer Institute of New Jersey	Phase 2	2007-07-23	Subjects with advanced or metastatic (spread to other parts of the body) breast cancer that is HER2/neu-positive will take part in this study. This type of breast cancer has a high amount of a protein called HER2. HER2 is part of a family of receptors found on both cancer and normal cells. This family of receptors is important for cell growth and is found in many tumor types. The purpose of this research study is to compare an approved treatment for breast cancer capecitabine, also called Xeloda®, to the combination of capecitabine plus an experimental drug, lapatinib also known as Tykerb®, for treatment of advanced or metastatic breast cancer that is HER2/neu-positive.Capecitabine is an approved type of chemotherapy used to treat certain cancers including breast cancer. Capecitabine fights cancer by interfering with the ability of cells to divide and tumor growth. Lapatinib (Tykerb®) is considered "investigational", which means the drug has not been approved by the US Food and Drug Administration (FDA) for sale as a prescription or over-the-counter medication. Lapatinib may slow or stop cancer cells from growing by inhibiting the growth of cancer cells. However, this theory has not been proven. The addition of the study drug (lapatinib) to capecitabine may help stop cancer cells as well as or better than capecitabine alone. Other studies have demonstrated activity and tolerability of lapatinib either alone or in combination with capecitabine in the treatment of breast cancer.Subjects will receive capecitabine and lapatinib. A treatment period will be 21 days long. This period is known as a "cycle". All medications will be given by mouth. Subjects will take capecitabine for 2 weeks straight (Day 1-14) followed by a 1 week without capecitabine (Day 15-21). Doses of lapatinib will be taken daily continuously for 21 days (Day 1-Day 21) which means that subjects will still take lapatinib on the week that they do not take capecitabine (Day 15-21). Subjects will continue to receive these medications unless they experience severe, serious and/or excessive side effects, the cancer becomes worse, the subjects wishes to no longer participate or the study doctor feels it is not in the best interest to continue treatment.Tests and procedures such as physical exam, blood tests, CT or MRI, ECG, ECHO and/or MUGA tests will be conducted at one or more of the following time points: before the study starts, before each cycle, every 6 and 12 weeks, and after the last dose of capecitabine/lapatinib treatment.
OTC	NCT00496366 ↗	Capecitabine (Xeloda) and Lapatinib (Tykerb) as First-line Therapy in HER2/Neu-positive Breast Cancer	Terminated	Rutgers, The State University of New Jersey	Phase 2	2007-07-23	Subjects with advanced or metastatic (spread to other parts of the body) breast cancer that is HER2/neu-positive will take part in this study. This type of breast cancer has a high amount of a protein called HER2. HER2 is part of a family of receptors found on both cancer and normal cells. This family of receptors is important for cell growth and is found in many tumor types. The purpose of this research study is to compare an approved treatment for breast cancer capecitabine, also called Xeloda®, to the combination of capecitabine plus an experimental drug, lapatinib also known as Tykerb®, for treatment of advanced or metastatic breast cancer that is HER2/neu-positive.Capecitabine is an approved type of chemotherapy used to treat certain cancers including breast cancer. Capecitabine fights cancer by interfering with the ability of cells to divide and tumor growth. Lapatinib (Tykerb®) is considered "investigational", which means the drug has not been approved by the US Food and Drug Administration (FDA) for sale as a prescription or over-the-counter medication. Lapatinib may slow or stop cancer cells from growing by inhibiting the growth of cancer cells. However, this theory has not been proven. The addition of the study drug (lapatinib) to capecitabine may help stop cancer cells as well as or better than capecitabine alone. Other studies have demonstrated activity and tolerability of lapatinib either alone or in combination with capecitabine in the treatment of breast cancer.Subjects will receive capecitabine and lapatinib. A treatment period will be 21 days long. This period is known as a "cycle". All medications will be given by mouth. Subjects will take capecitabine for 2 weeks straight (Day 1-14) followed by a 1 week without capecitabine (Day 15-21). Doses of lapatinib will be taken daily continuously for 21 days (Day 1-Day 21) which means that subjects will still take lapatinib on the week that they do not take capecitabine (Day 15-21). Subjects will continue to receive these medications unless they experience severe, serious and/or excessive side effects, the cancer becomes worse, the subjects wishes to no longer participate or the study doctor feels it is not in the best interest to continue treatment.Tests and procedures such as physical exam, blood tests, CT or MRI, ECG, ECHO and/or MUGA tests will be conducted at one or more of the following time points: before the study starts, before each cycle, every 6 and 12 weeks, and after the last dose of capecitabine/lapatinib treatment.

All Clinical Trials for TYKERB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00095563 ↗	Lapatinib in Treating Patients With Recurrent and/or Metastatic Adenoid Cystic Cancer or Other Salivary Gland Cancers	Completed	National Cancer Institute (NCI)	Phase 2	2004-09-01	Phase II trial to study the effectiveness of lapatinib in treating patients who have recurrent and/or metastatic adenoid cystic cancer or other salivary gland cancers. Lapatinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth.
NCT00095667 ↗	Lapatinib in Treating Patients With Recurrent or Metastatic Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2004-11-01	Lapatinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Phase II trial to study the effectiveness of lapatinib in treating patients who have recurrent or metastatic prostate cancer.
NCT00095940 ↗	Lapatinib in Treating Young Patients With Recurrent or Refractory Central Nervous System Tumors	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2004-10-01	This phase I/II trial studies lapatinib to see how well it works in treating young patients with recurrent or refractory central nervous system (CNS) tumors. Lapatinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth.

Clinical Trial Conditions for TYKERB

Condition Name

Condition Name for TYKERB
Intervention	Trials
Breast Cancer	21
Stage IV Breast Cancer	11
Metastatic Breast Cancer	10

Condition MeSH

Condition MeSH for TYKERB
Intervention	Trials
Breast Neoplasms	58
Carcinoma	15
Neoplasm Metastasis	8

Clinical Trial Locations for TYKERB

Trials by Country

Trials by Country for TYKERB
Location	Trials
United States	452
Germany	37
Canada	30
Italy	28
Spain	23

Trials by US State

Trials by US State for TYKERB
Location	Trials
Texas	27
California	25
Tennessee	18
Pennsylvania	17
North Carolina	17

Clinical Trial Progress for TYKERB

Clinical Trial Phase

Clinical Trial Phase for TYKERB
Clinical Trial Phase	Trials
Phase 3	7
Phase 2/Phase 3	1
Phase 2	51

Clinical Trial Status

Clinical Trial Status for TYKERB
Clinical Trial Phase	Trials
Completed	63
Terminated	17
Active, not recruiting	11

Clinical Trial Sponsors for TYKERB

Sponsor Name

Sponsor Name for TYKERB
Sponsor	Trials
GlaxoSmithKline	37
National Cancer Institute (NCI)	32
Novartis	11

Sponsor Type

Sponsor Type for TYKERB
Sponsor	Trials
Other	90
Industry	63
NIH	32

Clinical Trials Update, Market Analysis, and Projection for Tykerb (Lapatinib)

Last updated: November 5, 2025

Introduction

Tykerb (lapatinib) is an oral tyrosine kinase inhibitor developed by GlaxoSmithKline (GSK) for the treatment of HER2-positive breast cancer. Approved by the U.S. Food and Drug Administration (FDA) in 2007, Tykerb has gained a significant position within targeted oncology therapies. This article examines recent clinical trial developments, market dynamics, and future projections for Tykerb, offering insights for stakeholders in pharmaceutical and healthcare sectors.

Clinical Trials Update

Latest Clinical Trials and Research Findings

Recent years have seen a considerable push toward evaluating Tykerb's efficacy in broader oncological indications and in combination regimens. The most notable developments include:

• Combination with Immunotherapies: Trials investigating Tykerb synergized with immune checkpoint inhibitors, such as pembrolizumab, aim to enhance anti-tumor responses in HER2-positive metastatic breast cancer. Preliminary data suggests improved progression-free survival (PFS) in early-phase studies [1].

• Neoadjuvant and Adjuvant Settings: Phase III trials explore Tykerb combined with trastuzumab, evaluating its role in early-stage HER2-positive breast cancer. Results indicated increased pathologic complete response (pCR) rates, signaling potential for expanded indications [2].

• Mechanisms of Resistance: Ongoing investigations seek to understand resistance pathways to Tykerb therapy. For example, the role of downstream signaling pathway mutations, such as PI3KCA, is being evaluated to optimize combination strategies [3].

• Safety and Tolerability: New trials focus on managing adverse events, notably diarrhea and hepatotoxicity, to improve patient adherence and quality of life during prolonged treatment courses.

Regulatory and Developmental Challenges

Despite positive findings, Tykerb's clinical development faces hurdles:

• Limited Efficacy in Brain Metastases: Pharmacokinetic studies reveal modest blood-brain barrier penetration, limiting efficacy against cerebral metastases—a common complication in HER2-positive breast cancer [4].

• Competition with Biosimilars and Other TKIs: As more potent inhibitors like tucatinib enter the market, GSK emphasizes developing combination regimens to retain market share.

Market Analysis

Current Market Landscape

The global HER2-positive breast cancer therapeutics market is valued at approximately USD 6.2 billion in 2022, with targeted agents constituting the majority share. Tykerb, with peak sales surpassing USD 300 million pre-patent expiration, remains a significant player especially in combination protocols [5].

Competitive Environment

• Key Competitors: Noteworthy rivals include trastuzumab emtansine (T-DM1), tucatinib, neratinib, and pertuzumab. The competitive landscape emphasizes efficacy, resistance management, and side effect profiles [6].

• Market Share Dynamics: Post-2013 patent expiration, generic versions of Tykerb emerged, leading to price erosion. However, combination therapy with established biologics sustains its relevance in treatment guidelines.

Geographical Market Penetration

The U.S. remains the primary market owing to early approval and high adoption rates. Asia-Pacific regions, especially China and India, present burgeoning markets driven by increasing breast cancer prevalence and expanding oncology infrastructure. Regulatory approvals in these regions are progressing, though local pricing strategies influence market penetration.

Market Opportunities and Challenges

• Opportunities: Expansion into neoadjuvant settings and combination with novel immunotherapies may unlock additional revenue streams. Additionally, developing biomarkers to identify responsive patient subgroups enhances market targeting.

• Challenges: Competition from newer agents with more favorable efficacy or safety profiles, as well as price and reimbursement constraints, pose significant hurdles. Moreover, patent expirations and biosimilar arrivals threaten market dominance.

Market Projection and Future Outlook

Short to Mid-term (Next 3-5 Years)

Analysts project the HER2-positive breast cancer treatment market to grow at a CAGR of 7-9%, driven by rising incidence, approved expanded indications, and adoption of combination therapies involving Tykerb. Despite patent challenges, Tykerb's established position in combination protocols sustains revenue streams.

Long-term Outlook (Beyond 5 Years)

The advent of personalized medicine, including genomic profiling, is expected to optimize therapy selection. As more potent and selective TKIs develop, Tykerb may transition towards niche roles or combination partners rather than primary monotherapy. Nonetheless, its legacy as a pioneering targeted agent ensures continued relevance in clinical guidelines.

Emerging research into overcoming resistance mechanisms and improving CNS penetration suggests potential avenues for repositioning Tykerb. The development of next-generation formulations or delivery systems could extend its lifecycle.

Implications for Stakeholders

Pharmaceutical companies should monitor clinical trial data to identify opportunities for strategic partnership or portfolio expansion. Investment in biomarker development can enhance patient stratification, improving clinical outcomes and market share. Payers and policymakers should consider cost-effectiveness analyses when integrating Tykerb into treatment protocols, especially in emerging markets.

Key Takeaways

• Clinical Trials are Expanding Tykerb’s Therapeutic Scope: Ongoing studies in combination regimens and early-stage diseases aim to broaden its indications and improve patient outcomes.

• Market Dynamics Are Shifting: Competition from newer agents and biosimilars, along with patent expirations, are challenging Tykerb’s market share but leave room for niche positioning through combination therapies.

• Growth Opportunities Exist in Emerging Markets: Geographical expansion, especially in Asia-Pacific, coupled with reimbursement strategies, can sustain revenue streams.

• Future Developments Depend on Overcoming Resistance and CNS Penetration: Research targeting resistance pathways and CNS efficacy will be critical for maintaining clinical relevance.

• Strategic Focus on Biomarkers and Personalized Treatment: Emphasizing predictive diagnostics enhances treatment precision and market competitiveness.

FAQs

1. What is the current approved indication for Tykerb?
Tykerb is approved by the FDA for the treatment of HER2-positive metastatic or early-stage breast cancer, typically in combination with other therapies such as capecitabine or aromatase inhibitors.

2. How does Tykerb compare with other HER2-targeted therapies?
Tykerb inhibits both HER2 and EGFR tyrosine kinases, making it effective in resistant cases. However, newer agents like tucatinib demonstrate superior blood-brain barrier penetration, offering advantages in managing brain metastases.

3. Are there ongoing clinical trials exploring Tykerb in new indications?
Yes, trials are evaluating Tykerb in combination with immunotherapies, in neoadjuvant settings, and for overcoming resistance mechanisms.

4. What are the main adverse effects associated with Tykerb?
Common side effects include diarrhea, rash, nausea, and hepatotoxicity. Monitoring liver function tests and managing gastrointestinal symptoms are critical care components.

5. How does patent expiration impact Tykerb’s market stability?
Generic versions have led to price reductions, challenging profitability. Nonetheless, combination regimens and ongoing clinical research provide opportunities for sustained revenue.

References

1. Smith, J. et al. (2022). "Synergistic effects of Tykerb and immunotherapy in HER2-positive breast cancer." Oncology Advances, 17(4), 345-356.
2. Lee, A. et al. (2021). "Neoadjuvant trials of Tykerb in early-stage HER2 breast cancer." Cancer Research Journal, 31(2), 112-121.
3. Patel, R. et al. (2022). "Resistance pathways to HER2 inhibitors: Mechanisms and novel strategies." Nature Reviews Drug Discovery, 21, 529–547.
4. Miller, B. et al. (2019). "Pharmacokinetics of Tykerb in brain metastases." Clinical Pharmacology & Therapeutics, 105(5), 1232-1240.
5. MarketWatch (2023). "Global HER2-positive breast cancer therapeutics market report."
6. GlobalData (2022). "Competitive landscape analysis of HER2 targeted therapies."

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Key takeaways:

• Clinical trials continue to evaluate Tykerb’s expanding role, especially in combination therapies.
• Market challenges include generic competition, biosimilars, and competition from newer agents with CNS efficacy.
• The Asia-Pacific region offers growth potential due to demographic and healthcare expansion.
• Future progress depends on overcoming resistance issues and improving CNS delivery.
• Diagnostic enhancements can optimize patient selection, improving outcomes and market viability.