CLINICAL TRIALS PROFILE FOR EFUDEX

505(b)(2) Clinical Trials for Efudex

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	NCT02019355 ↗	Actinic Keratosis Study	Completed	Washington University School of Medicine	Early Phase 1	2013-10-01	The main purpose of this study is to determine the effectiveness of a new combination therapy for actinic keratosis. This study investigates a new indication for an FDA-approved topical medication, calcipotriol, for treatment of actinic keratosis, including how well it works and how safe it is when used in combination with the standard of care medication (5-fluorouracil) for the skin condition.
New Indication	NCT02019355 ↗	Actinic Keratosis Study	Completed	Washington University School of Medicine	Early Phase 1	2013-10-01	The main purpose of this study is to determine the effectiveness of a new combination therapy for actinic keratosis. This study investigates a new indication for an FDA-approved topical medication, calcipotriol, for treatment of actinic keratosis, including how well it works and how safe it is when used in combination with the standard of care medication (5-fluorouracil) for the skin condition.

All Clinical Trials for Efudex

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002525 ↗	Perioperative Chemotherapy in Treating Patients With Colon Cancer That Can Be Surgically Removed	Terminated	American College of Surgeons	Phase 3	1993-08-01	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 and giving drugs in different ways may kill more tumor cells. It is not yet known if surgery is more effective with or without chemotherapy for colon cancer. PURPOSE: Randomized phase III trial to evaluate whether perioperative 5-Fluorouracil (5-FU) chemotherapy after curative resection could improve overall survival and disease-free survival in patients with Duke's B3 or C colon cancer.
NCT00002525 ↗	Perioperative Chemotherapy in Treating Patients With Colon Cancer That Can Be Surgically Removed	Terminated	Cancer and Leukemia Group B	Phase 3	1993-08-01	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 and giving drugs in different ways may kill more tumor cells. It is not yet known if surgery is more effective with or without chemotherapy for colon cancer. PURPOSE: Randomized phase III trial to evaluate whether perioperative 5-Fluorouracil (5-FU) chemotherapy after curative resection could improve overall survival and disease-free survival in patients with Duke's B3 or C colon cancer.
NCT00002525 ↗	Perioperative Chemotherapy in Treating Patients With Colon Cancer That Can Be Surgically Removed	Terminated	National Cancer Institute (NCI)	Phase 3	1993-08-01	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 and giving drugs in different ways may kill more tumor cells. It is not yet known if surgery is more effective with or without chemotherapy for colon cancer. PURPOSE: Randomized phase III trial to evaluate whether perioperative 5-Fluorouracil (5-FU) chemotherapy after curative resection could improve overall survival and disease-free survival in patients with Duke's B3 or C colon cancer.
NCT00002525 ↗	Perioperative Chemotherapy in Treating Patients With Colon Cancer That Can Be Surgically Removed	Terminated	NSABP Foundation Inc	Phase 3	1993-08-01	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 and giving drugs in different ways may kill more tumor cells. It is not yet known if surgery is more effective with or without chemotherapy for colon cancer. PURPOSE: Randomized phase III trial to evaluate whether perioperative 5-Fluorouracil (5-FU) chemotherapy after curative resection could improve overall survival and disease-free survival in patients with Duke's B3 or C colon cancer.

Clinical Trial Conditions for Efudex

Condition Name

Condition Name for Efudex
Intervention	Trials
Breast Cancer	7
Colorectal Cancer	6
Pancreatic Cancer	5
Recurrent Rectal Cancer	4

Condition MeSH

Condition MeSH for Efudex
Intervention	Trials
Adenocarcinoma	12
Colorectal Neoplasms	11
Carcinoma	11
Pancreatic Neoplasms	11

Clinical Trial Locations for Efudex

Trials by Country

Trials by Country for Efudex
Location	Trials
United States	370
Canada	21
Japan	18
Spain	15
Belgium	13

Trials by US State

Trials by US State for Efudex
Location	Trials
Texas	34
California	18
Pennsylvania	15
New York	15
Michigan	15

Clinical Trial Progress for Efudex

Clinical Trial Phase

Clinical Trial Phase for Efudex
Clinical Trial Phase	Trials
Phase 3	12
Phase 2/Phase 3	1
Phase 2	33

Clinical Trial Status

Clinical Trial Status for Efudex
Clinical Trial Phase	Trials
Completed	41
Terminated	11
Recruiting	8

Clinical Trial Sponsors for Efudex

Sponsor Name

Sponsor Name for Efudex
Sponsor	Trials
National Cancer Institute (NCI)	31
M.D. Anderson Cancer Center	25
Genentech, Inc.	5

Sponsor Type

Sponsor Type for Efudex
Sponsor	Trials
Other	65
Industry	42
NIH	31

EFUDEX: Clinical Trials, Market Analysis, and Projections

Introduction to EFUDEX

EFUDEX, also known as fluorouracil, is a topical chemotherapeutic agent widely used in the treatment of actinic keratosis and superficial basal cell carcinoma. Here, we will delve into the clinical trials, market analysis, and future projections for EFUDEX.

Clinical Trials Overview

Phase 3 Trials for Tolak (Fluorouracil) Cream 4%

Clinical trials for EFUDEX, particularly those involving Tolak (fluorouracil) Cream 4%, have provided significant insights into its efficacy and safety. Two key Phase 3 trials, HD-FUP3B-048 and HP-FU3S-049, were conducted to evaluate the efficacy and safety of once-daily applications of Tolak Cream 4% compared to Efudex 5% cream and vehicle controls.

• HD-FUP3B-048: This study compared the efficacy and safety of Tolak Cream 4% applied once daily over four weeks against Efudex 5% cream applied twice daily, as well as against vehicle controls. Although noninferiority to Efudex was not demonstrated, statistical superiority to the vehicle was established[1].
• HP-FU3S-049: This trial evaluated the efficacy and safety of once-daily applications of Tolak Cream 4% against its vehicle. Statistical superiority to the vehicle was again demonstrated in this study[1].

Long-Term Safety Data

Study 50, a long-term follow-up study, supported the safety data obtained from the pivotal trials. This study involved 310 subjects and showed that the adverse events observed were similar to those seen in the Phase 3 trials, primarily related to application site reactions. The discontinuation rate due to adverse events was lower in this long-term study (3%) compared to the Phase 3 studies (15%)[1].

Market Analysis

Current Market Trends

The EFUDEX cream market is experiencing significant growth driven by several key factors:

• Increasing Awareness: Growing awareness about skin cancer and precancerous conditions, such as actinic keratosis, is leading to higher adoption rates of EFUDEX cream[2].
• Technological Advancements: Improvements in dermatological products and rising healthcare expenditure are contributing to market expansion[2].
• Aging Population: The increasing prevalence of skin disorders among an aging population is another driving factor[2].

Market Size and Growth Projections

The EFUDEX cream market is anticipated to witness substantial growth:

• The global EFUDEX cream market is projected to grow at a compound annual growth rate (CAGR) of 10.48% from 2024 to 2031, reaching a valuation of USD 37.32 billion by 2031 from USD 18.58 billion in 2024[2].

Regional Analysis

• North America: This region, particularly the United States and Canada, is a significant player in the global EFUDEX cream market, driven by a robust economy and technological advancements[2].
• Europe: Europe is another major region, characterized by a mature market with well-established infrastructure and consumer preferences[2].
• Asia-Pacific: This region is rapidly growing, driven by countries like China, Japan, India, and South Korea, with a large population and rising disposable income[2].
• Latin America and Middle East & Africa: These regions present opportunities and challenges, with countries like Brazil, Mexico, and South Africa showing promising growth potential[2].

Market Segments and Competitors

Topical/Drugs Segment

The nucleoside metabolic inhibitors segment, which includes EFUDEX, dominates the market. The strong commercial performance of products like Fluroplex, Carac, and EFUDEX, along with higher demand for topical formulations, drives this segment's growth. EFUDEX, specifically 5-fluorouracil, held a 32.28% share of the actinic keratosis (AK) drugs market in the U.S. in 2023, due to its high efficacy and lower chances of non-response[3].

Key Players

Major companies in the EFUDEX cream market include:

• Bausch Health
• Mayne Pharma

These companies are involved in various strategies such as mergers and acquisitions, and research and development to enhance their market positions and product portfolios[2].

Future Projections and Growth Drivers

Technological Advancements

Ongoing research and development activities aimed at enhancing the formulation and efficacy of EFUDEX cream are expected to drive future market expansion. For instance, clinical studies like the one initiated by Biofrontera Inc. in 2022 to evaluate the safety of new treatments for actinic keratosis will contribute to segment growth[3].

Increasing Awareness and Early Diagnosis

Awareness campaigns and early diagnosis initiatives are expected to continue driving the demand for EFUDEX cream globally. Partnerships, such as Almirall's collaboration with Euromelanoma, are crucial in raising awareness about skin conditions and promoting the use of effective treatments like EFUDEX[3].

Market Consolidation

The market is witnessing a wave of mergers and acquisitions as companies seek to consolidate their market positions, expand their product portfolios, and leverage synergies to drive growth and competitiveness. These strategic moves provide access to new technologies, intellectual property, and talent, enabling companies to innovate faster and stay ahead of market trends[2].

Key Takeaways

• Clinical Trials: EFUDEX has demonstrated efficacy and safety in various clinical trials, including Phase 3 studies and long-term follow-up.
• Market Growth: The EFUDEX cream market is projected to grow significantly, driven by increasing awareness, technological advancements, and an aging population.
• Regional Dynamics: North America, Europe, and Asia-Pacific are key regions driving the market growth.
• Competitive Landscape: Major players like Bausch Health and Mayne Pharma are focusing on R&D and strategic consolidations to maintain market leadership.
• Future Projections: Ongoing research, awareness campaigns, and market consolidation are expected to fuel the market's growth.

FAQs

1. What is the current market size of the EFUDEX cream market?

   • The global EFUDEX cream market was valued at USD 18.58 billion in 2024[2].

2. What is the projected growth rate of the EFUDEX cream market?

   • The market is anticipated to grow at a CAGR of 10.48% from 2024 to 2031[2].

3. Which regions are driving the growth of the EFUDEX cream market?

   • North America, Europe, and Asia-Pacific are significant regions driving the market growth[2].

4. What are the key factors driving the demand for EFUDEX cream?

   • Increasing awareness about skin cancer, technological advancements, rising healthcare expenditure, and an aging population are key drivers[2].

5. Who are the major players in the EFUDEX cream market?

   • Major players include Bausch Health and Mayne Pharma[2].

Sources

1. FDA Clinical Review: "NDA 22-259 Tolak (fluorouracil) Cream, 4%" - accessdata.fda.gov
2. OpenPR Market Report: "Efudex Cream Market Size, Share and Forecast By Key Players" - openPR.com
3. Nova One Advisor Report: "U.S. Actinic Keratosis Treatment Market Size, Share, Trends" - novaoneadvisor.com