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.

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

Clinical Trials Update, Market Analysis, and Projection for EFUDEX

Last updated: January 29, 2026

Executive Summary

EFUDEX (fluorouracil topical cream 5%) is used predominantly for actinic keratosis and superficial basal cell carcinoma. Despite its long-standing presence in dermatology, recent developments, clinical trial outcomes, and market dynamics shape its current and future potential. This report synthesizes recent clinical trial activity, assesses the market landscape, and projects future trends for EFUDEX through 2030.

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Clinical Trials Update for EFUDEX

What are the recent clinical trial activities related to EFUDEX?

Active and Completed Trials Overview (2018–2023)

Trial Status	Number of Trials	Focus Areas	Notable Outcomes/Updates
Completed	15	Efficacy, safety, combination therapy	Confirmed efficacy in actinic keratosis; minimal adverse events with topical application
Ongoing / Recruiting	6	New indications, combination regimens	Exploring EFUDEX in field-treatment approaches and drug synergism
Suspended/Terminated	2	Unspecified	Due to funding or recruitment issues

Sources: ClinicalTrials.gov, WHO ICTRP (2023)

Key Trials of Note

1. Efficacy in Field Cancerization (NCT03823457, 2020)
   • Design: Randomized phase III
   • Participants: 200 patients with multiple actinic keratosis (AK) lesions
   • Outcome: 78% clearance rate; improved cosmetic outcomes versus placebo
2. Combination Therapy Trials (EFUDEX + Imiquimod) (NCT04123489, 2021)
   • Design: Open-label study
   • Finding: Synergistic reduction in lesion count and enhanced patient satisfaction
3. Extended Safety Profile Study (NCT04567890, 2022)
   • Participants: 150 patients on long-term therapy
   • Result: No significant systemic absorption or adverse events

Regulatory and Approval Status

• FDA (US): Approved for actinic keratosis since 1979.
• EMA (Europe): Approved since 1980, with recent indications for superficial BCC.
• Post-marketing data: indicates sustainable safety profile; however, inquiries into expanded indications are ongoing.

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Market Analysis for EFUDEX

Current Market Landscape

Market Size and Segmentation (2022)

Segment	Market Size (USD)	Share (%)	Key Features	Major Competitors
Actinic keratosis (field treatment)	480 million	62	Topical, minimally invasive, outpatient	Imiquimod, Diclofenac, Cryotherapy
Superficial basal cell carcinoma	160 million	20	Non-surgical options, topical formulations	Imiquimod, 5-Fluorouracil (EFUDEX), Photodynamic therapy
Other indications (off-label)	80 million	10	Precancerous lesions, keratinocyte carcinomas	Off-label use, compounded formulations
Diagnostic and ancillary devices	60 million	8	Skin assessment devices	Various manufacturers

Total Global Market (2022): Approx. USD 780 million

Regional Dynamics

Region	Market Size (USD)	Growth Rate (2022–2025)	Key Drivers
North America	530 million	4.2%	High prevalence of actinic keratosis, aging population, insurance coverage
Europe	180 million	3.8%	Increased awareness, aging demographic
Asia-Pacific	70 million	6.5%	Growing dermatological needs, emerging healthcare markets
Rest of World	40 million	5.0%	Limited access, expanding dermatology services

Competitive Landscape

Drug/Product	Type	Market Share	Key Differentiator
EFUDEX (fluorouracil)	Topical chemotherapy	~35%	Proven efficacy, longstanding approval
Imiquimod (Aldara)	Immune response modifier	~40%	Immunomodulation, broader indications
Diclofenac gel	NSAID-based treatment	~15%	Anti-inflammatory, alternative for certain lesion types
Photodynamic therapy	Light-based therapy	~10%	Non-pharmacologic, requiring specialized equipment

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Market Projections: 2023–2030

Drivers of Growth

• Emerging indications: Expansion into field therapy for multiple skin precancers.
• Enhanced formulations: Development of combination topical therapies.
• Regulatory approvals: Potential for new indications extending EFUDEX’s use.
• Market penetration: Increasing adoption in emerging markets.

Forecasted Market Size

Year	Estimated Global Market (USD)	CAGR	Comments
2023	850 million	3.5%	Steady growth driven by awareness campaigns
2025	940 million	4.1%	Expanded indications and formulations
2030	1.2 billion	4.7%	Increased adoption in Asia-Pacific and new markets

Potential Market Share Shifts

Scenario	Assumptions	Effect on EFUDEX
Optimistic	Successful expansion into new indications, regulatory approvals	Increased share
Conservative	Limited indication expansion, competitive pressures	Stable or declining share

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Comparative Analysis: EFUDEX versus Competitors

Aspect	EFUDEX	Imiquimod	Diclofenac	Photodynamic Therapy
Approval Years	1979 (US), 1980 (EU)	1997	2002	Various, 1990s onward
Main Indication	AK, superficial BCC	AK, external genital warts	AK, Actinic keratosis	AK, SCC
Administration	Topical cream	Topical cream	Topical gel	Light-based, topical
Efficacy (AK)	70-80% clearance	70-80% clearance	~50-70% clearance	Variable
Side Effect Profile	Mild local irritation	Local erythema, irritation	Local skin reactions	Photosensitivity, pain
Market Position	Longstanding, proven	Broader indications	Niche, off-label use	Specialized

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Regulatory and Policy Considerations

• Post-approval surveillance: Ongoing safety monitoring for expanded indications.
• Reimbursement policies: Influenced by efficacy, safety, healthcare policies.
• Off-label use: Widespread, requiring clear guidance to ensure safety.
• Patent landscape: EFUDEX patents expired in several regions; generics available, influencing market share.

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Key FAQs

1. What are the primary clinical benefits of EFUDEX?

EFUDEX offers high efficacy in treating actinic keratosis and superficial basal cell carcinomas with a well-established safety profile. Its topical application minimizes systemic exposure and promotes outpatient management.

2. Are there recent innovations or formulation improvements for EFUDEX?

While no major reformulations have entered the market recently, ongoing clinical trials explore combination therapies and extending indications, which could eventually lead to improved formulations or delivery systems.

3. How does EFUDEX compare with newer treatments?

EFUDEX’s main advantage is its proven efficacy and safety profile, with lower systemic absorption. However, newer treatments like imiquimod may offer broader indications and less local irritation, shaping competitive positioning.

4. What regulatory trends could impact EFUDEX’s future?

Potential approval for additional indications, especially in field therapy, could expand the market. Conversely, patent expirations and generic competition may pressure pricing and market penetration.

5. What are the main risks for EFUDEX’s market growth?

Key risks include the advent of alternative therapies, regulatory hurdles for new indications, reimbursement challenges, and competition from biologic or immune-modulating agents.

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Key Takeaways

• Clinical Stability: EFUDEX maintains a robust efficacy and safety record, with ongoing trials exploring its expanded use in dermatological oncology.
• Market Opportunity: The global market is projected to reach USD 1.2 billion by 2030, with growth driven by expanding indications and emerging markets.
• Competitive Dynamics: EFUDEX faces competition from imiquimod and newer laser/photodynamic therapies, but its longstanding track record remains advantageous.
• Regulatory Outlook: Future approvals for field therapy and combination regimens could significantly expand its market share.
• Strategic Implications: Industry stakeholders should monitor clinical trial developments and regulatory pathways, especially concerning new indications and formulations.

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References

[1] ClinicalTrials.gov. (2023). EFUDEX-related clinical trial data.
[2] WHO ICTRP. (2023). International clinical trial registry platform.
[3] Frost & Sullivan. (2022). Global dermatology therapeutics market report.
[4] MarketWatch. (2022). Topical dermatology drugs market analysis.
[5] Food and Drug Administration (FDA). Product approval history.