CLINICAL TRIALS PROFILE FOR GEFITINIB

505(b)(2) Clinical Trials for Gefitinib

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	NCT02353741 ↗	Concurrent EGFR-TKIs and Thoracic Radiation Therapy in Active EGFR Mutation for 1st Line Treatment of Stage IV NSCLC	Terminated	Xinqiao Hospital of Chongqing	Phase 2	2015-04-01	This single-arm phase II study aims to study the efficacy of a possible first line treatment that combines epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKIs) with concurrent thoracic radiation therapy for stage IV non-small cell lung cancer (NSCLC) with active EGFR mutation, as well as assessing PFS, OS, tumor response, etc. to verify that this new combinational therapy can benefit short-term and long-term survival of the patients with advanced NSCLC.

All Clinical Trials for Gefitinib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00005806 ↗	Combination Chemotherapy in Treating Patients With Advanced Non-Small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 1	1999-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase I trial to study the effectiveness of combination chemotherapy in treating patients who have advanced non-small cell lung cancer.
NCT00005806 ↗	Combination Chemotherapy in Treating Patients With Advanced Non-Small Cell Lung Cancer	Completed	Memorial Sloan Kettering Cancer Center	Phase 1	1999-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase I trial to study the effectiveness of combination chemotherapy in treating patients who have advanced non-small cell lung cancer.
NCT00006048 ↗	ZD 1839 Plus Combination Chemotherapy in Treating Patients With Non-Small Cell Lung Cancer	Unknown status	AstraZeneca	Phase 3	2000-05-01	RATIONALE: Some tumors need growth factors produced by the body's white blood cells to keep growing. ZD 1839 may interfere with the growth factor and stop the tumor from growing. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether chemotherapy is more effective with or without ZD 1839 for non-small cell lung cancer. PURPOSE: Randomized phase III trial to compare the effectiveness of combination chemotherapy with or without ZD 1839 in treating patients who have stage IIIB or stage IV non-small cell lung cancer.
NCT00006049 ↗	ZD 1839 Plus Chemotherapy in Treating Patients With Non-Small Cell Lung Cancer	Unknown status	AstraZeneca	Phase 3	2000-05-01	RATIONALE: Some tumors need growth factors produced by the body's white blood cells to keep growing. ZD 1839 may interfere with the growth factor and stop the tumor from growing. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether combination chemotherapy is more effective with or without ZD 1839 for non-small cell lung cancer. PURPOSE: Randomized phase III trial to compare the effectiveness of combination chemotherapy with or without ZD 1839 in treating patients who have stage III or stage IV non-small cell lung cancer.
NCT00012337 ↗	ZD 1839 in Treating Patients With Metastatic Kidney Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2001-01-01	RATIONALE: Some tumors need growth factors produced by the body's white blood cells to keep growing. Drugs such as ZD 1839 may interfere with the growth factors and cause tumor cells to die. PURPOSE: Phase II trial to study the effectiveness of ZD 1839 in treating patients who have metastatic kidney cancer.

Clinical Trial Conditions for Gefitinib

Condition Name

Condition Name for Gefitinib
Intervention	Trials
Non-small Cell Lung Cancer	56
Lung Cancer	39
Non Small Cell Lung Cancer	24
Carcinoma, Non-Small-Cell Lung	19

Condition MeSH

Condition MeSH for Gefitinib
Intervention	Trials
Carcinoma, Non-Small-Cell Lung	203
Lung Neoplasms	171
Adenocarcinoma	29
Carcinoma	25

Clinical Trial Locations for Gefitinib

Trials by Country

Trials by Country for Gefitinib
Location	Trials
United States	515
China	167
Japan	92
Korea, Republic of	90
Italy	88

Trials by US State

Trials by US State for Gefitinib
Location	Trials
New York	40
Texas	33
California	30
Massachusetts	28
Pennsylvania	24

Clinical Trial Progress for Gefitinib

Clinical Trial Phase

Clinical Trial Phase for Gefitinib
Clinical Trial Phase	Trials
Phase 4	11
Phase 3	53
Phase 2/Phase 3	9

Clinical Trial Status

Clinical Trial Status for Gefitinib
Clinical Trial Phase	Trials
Completed	202
Unknown status	76
Terminated	36

Clinical Trial Sponsors for Gefitinib

Sponsor Name

Sponsor Name for Gefitinib
Sponsor	Trials
AstraZeneca	91
National Cancer Institute (NCI)	65
Sun Yat-sen University	13

Sponsor Type

Sponsor Type for Gefitinib
Sponsor	Trials
Other	424
Industry	197
NIH	68

Gefitinib: Clinical Trials, Market Analysis, and Projections

Introduction to Gefitinib

Gefitinib, marketed under the brand name Iressa, is a tyrosine kinase inhibitor (TKI) that targets the epidermal growth factor receptor (EGFR) tyrosine kinase. It is primarily used in the treatment of non-small cell lung cancer (NSCLC) with specific EGFR mutations. Here, we will delve into recent clinical trials, market analysis, and future projections for gefitinib.

Clinical Trials Update

Efficacy and Safety in EGFR-Mutant NSCLC with Brain Metastases

A recent phase 3 randomized clinical trial investigated the efficacy and safety of gefitinib plus chemotherapy compared to gefitinib alone in patients with untreated EGFR-mutant NSCLC and brain metastases. The trial, conducted in six centers in China from 2016 to 2021, enrolled 161 patients who were randomly assigned to receive either gefitinib plus chemotherapy (pemetrexed with platinum) or gefitinib alone[1].

Key findings from this trial include:

• Improved Survival Outcomes: Gefitinib plus chemotherapy significantly improved intracranial progression-free survival (PFS), overall PFS, and overall survival (OS) compared to gefitinib alone.
• Better Response Rates: The combination therapy had higher intracranial and overall objective response rates.
• Manageable Adverse Events: Although grade 3 or worse adverse events were more common in the combination group, they were generally manageable.

This study suggests that gefitinib plus chemotherapy could be a viable first-line treatment for patients with EGFR-mutant NSCLC and brain metastases[1].

Long-Term Benefits of Combination Therapy

Another randomized clinical trial confirmed that the benefits of adding chemotherapy to gefitinib were sustained over a five-year period. This trial showed that patients with EGFR-variant NSCLC who received gefitinib plus chemotherapy had improved PFS and OS compared to those receiving gefitinib alone. The hazard ratios for PFS and OS were 0.51 and 0.45, respectively, indicating a significant advantage for the combination therapy[4].

Market Analysis

Current Market Size and Growth

As of 2024, the global gefitinib market size is estimated to be USD 1541.2 million. This market is expected to grow at a compound annual growth rate (CAGR) of 8.50% from 2024 to 2031, reaching USD 2728.1 million by 2031[2].

Geographical Distribution

The market is segmented geographically, with North America holding the largest share, accounting for over 40% of the global revenue (USD 616.48 million in 2024). Europe and the Asia Pacific region follow, with market sizes of USD 462.36 million and USD 354.48 million, respectively, in 2024. These regions are expected to grow at CAGRs of 7.0%, 6.7%, and 10.5%, respectively, from 2024 to 2031[2].

Drivers of Market Growth

The growth of the gefitinib market is driven by several factors:

• Increasing Incidence of NSCLC: The rising number of NSCLC cases globally contributes to the demand for effective treatments like gefitinib.
• Advancements in Precision Medicine: The ability to identify specific EGFR mutations has made gefitinib a targeted and effective treatment option.
• Rising Healthcare Expenditure: Increased spending on healthcare, particularly in oncology, supports the adoption of premium-priced treatments like gefitinib[2].

Combination Therapies and Future Directions

Synergistic Approaches

The development of combination therapies involving gefitinib is a promising strategy to enhance treatment efficacy and overcome resistance mechanisms. For example, combining gefitinib with immune checkpoint inhibitors like pembrolizumab has shown promising results in certain NSCLC populations. These combination therapies target multiple pathways involved in cancer growth, potentially reducing adverse effects and extending the benefits to broader patient populations[2].

Regulatory and Clinical Context

Gefitinib was initially approved by the FDA in 2003 for the treatment of advanced NSCLC but was later withdrawn due to lack of confirmatory evidence. However, in 2015, it was reapproved for the initial treatment of patients with metastatic NSCLC whose tumors have specific EGFR mutations, along with a companion diagnostic test to identify eligible patients[3].

Key Takeaways

• Clinical Efficacy: Gefitinib plus chemotherapy has shown significant improvements in PFS and OS for patients with EGFR-mutant NSCLC, especially those with brain metastases.
• Market Growth: The global gefitinib market is expected to grow substantially, driven by increasing NSCLC incidence, advancements in precision medicine, and rising healthcare expenditure.
• Combination Therapies: The future of gefitinib treatment lies in combination therapies that target multiple cancer pathways, potentially offering better outcomes and reduced resistance.

FAQs

What is the current global market size of gefitinib?

The global gefitinib market size is estimated to be USD 1541.2 million as of 2024[2].

How is the gefitinib market expected to grow?

The gefitinib market is expected to grow at a CAGR of 8.50% from 2024 to 2031, reaching USD 2728.1 million by 2031[2].

What are the key drivers of the gefitinib market growth?

The key drivers include the increasing incidence of NSCLC, advancements in precision medicine, and rising healthcare expenditure[2].

What are the benefits of combining gefitinib with chemotherapy?

Combining gefitinib with chemotherapy has been shown to improve intracranial PFS, overall PFS, and OS in patients with EGFR-mutant NSCLC, especially those with brain metastases[1][4].

What is the regulatory status of gefitinib in the U.S.?

Gefitinib was reapproved by the FDA in 2015 for the initial treatment of patients with metastatic NSCLC whose tumors have specific EGFR mutations, along with a companion diagnostic test[3].

How does gefitinib work?

Gefitinib works by inhibiting the EGFR tyrosine kinase, thereby halting the growth and spread of cancer cells that depend on EGFR signaling pathways[2][3].