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.

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 (fluorouracil) Clinical Trials Update, Market Analysis, and Projection

Last updated: April 30, 2026

What is Efudex (fluorouracil) and how is it positioned today?

Efudex is the brand name for fluorouracil (5-FU) formulated for dermatologic use as a topical drug (commonly as a cream). It is used for actinic keratosis and other superficial cutaneous disorders where local inhibition of rapidly proliferating cells is needed.

Because Efudex is an established molecule with long market history, current commercially relevant activity is typically driven by:

• Line-extension type studies (new patient subsets, regimens, vehicle comparisons)
• Comparative effectiveness against other field-therapy agents (e.g., imiquimod, diclofenac, photodynamic therapy)
• Evidence refresh around outcomes such as lesion clearance and recurrence, including real-world adherence and tolerability patterns

What clinical trial signals exist for Efudex (fluorouracil topical)?

A current, decision-grade clinical trials update requires a closed list of active, completed, and recent publications tied to topical fluorouracil (Efudex brand) with dates, trial identifiers, endpoints, and results. No such complete, citable dataset is present in the available material for this response. Under the operating constraint, a complete and accurate update cannot be produced.

How is Efudex used clinically and what constraints matter for uptake?

Efudex uptake depends on four practical factors that shape physician choice and patient willingness to adhere:

1. Treatment field size and lesion count: outcomes and tolerability scale with the treated surface area.
2. Course length: patient burden increases with longer regimens, which impacts adherence.
3. Local skin reactions: erythema, irritation, and crusting can limit continued use.
4. Competition with procedural and newer field therapies: photodynamic therapy and other topical agents can shift prescribing patterns.

From a market-access perspective, topical 5-FU is often positioned as a cost-effective standard compared with newer branded field therapies, but the realized net price is constrained by:

• Generic competition for fluorouracil topical products in many markets
• Reimbursement rules tied to indications and documentation of lesion burden

What does the competitive landscape look like?

Efudex competes in a dermatology field-therapy setting that includes:

• Other topical anti-inflammatory/immune-modulating agents for actinic keratosis
• Topical cytotoxics and excipients with different dosing schedules
• Procedural therapies (including light-based approaches) that alter the convenience-versus-cost tradeoff

The practical competitive axis is not molecule novelty. It is experience, tolerability, speed of response, clinician familiarity, and total regimen burden.

How large is the Efudex market and what is the realistic growth path?

A market analysis with projection requires quantitative inputs:

• Total addressable market (TAM) for actinic keratosis and adjacent indications in key geographies
• Efudex-specific share drivers (formulation, pricing, channel mix)
• Recent unit/sales trends and documented demand effects

No such quantitative, citable dataset is available in the provided context for this response. Under the operating constraint, a complete and accurate market analysis and projection cannot be produced.

What would a projection model require for Efudex?

A decision-grade projection for an established topical product is typically built on:

• Indication-level incidence and eligible patient counts
• Re-treatment cycles and recurrence rates
• Switch rates between field therapies by tolerability and regimen burden
• Price erosion from generic entry and pack-level competitive pricing
• Payer rules and formulary placement dynamics

Without access to those numeric inputs and without citable trial and sales records, no defensible forecast can be generated.

What actionable insights can be stated from the drug’s profile?

Efudex market behavior is usually dominated by structural demand and access rather than clinical breakthroughs:

• Low innovation headroom: molecule maturity limits new label expansion probability
• High sensitivity to pricing and generic dynamics
• Strong dependence on guideline-based use for actinic keratosis and related superficial conditions
• Regimen adherence as a key determinant of real-world effectiveness, affecting net outcomes and continued prescribing

These are operational drivers, not a quantified market forecast.

Key Takeaways

• Efudex is an established topical fluorouracil (5-FU) therapy used primarily for superficial dermatologic conditions, most notably actinic keratosis.
• A decision-grade clinical trials update and market projection cannot be generated without a complete, citable set of current trial results and sales/market inputs tied to Efudex/topical fluorouracil.
• Commercial outcomes for Efudex are structurally driven by regimen burden, tolerability, guideline-driven prescribing, and generic/price pressure rather than late-stage innovation.

FAQs

1. What is Efudex used for?
   Efudex is used as a topical fluorouracil therapy for superficial skin disorders, with actinic keratosis as a core use case.

2. Is Efudex likely to have major late-stage clinical breakthroughs?
   For an established topical 5-FU product, meaningful growth is more likely to come from evidence refresh and regimen optimization than from new mechanism breakthroughs.

3. What drives patient adherence for topical fluorouracil?
   The intensity and duration of local skin reactions and the overall length of the treatment course are primary adherence drivers.

4. How does pricing affect Efudex demand?
   Generic competition and pack-level pricing typically pressure net pricing, shaping prescribing when payers and formularies guide selection.

5. What is the most relevant competition for Efudex in actinic keratosis?
   Other field therapies (topicals and procedural options) compete on total regimen burden, tolerability, and convenience as much as on efficacy.

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References

[1] Efudex (fluorouracil) prescribing information. FDA label repository.
[2] ClinicalTrials.gov database entries for topical fluorouracil / 5-FU in actinic keratosis and related dermatologic indications.