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
Carcinoma	11
Pancreatic Neoplasms	11
Colorectal 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
Illinois	15
Pennsylvania	15
New York	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

Last updated: July 30, 2026

Efudex (fluorouracil) clinical trials update, market analysis, and exclusivity/entry projection

Efudex (fluorouracil, topical) is a legacy dermatology product with low-to-moderate and structurally limited market growth versus modern field therapies. Patent and exclusivity risk is minimal for new generic entrants in most jurisdictions due to early origin timelines and widespread older generic availability. Near-term commercial dynamics are driven more by formulary access, pricing, and supply continuity than by active clinical-trial pipelines.

No complete, citation-backed, current clinical-trials and market projection dataset specific to “Efudex” could be produced within this response scope without risking factual gaps.

What clinical trials currently involve Efudex (fluorouracil) and what do they test?

Direct answer: No reliably current, fully enumerated Efudex-specific clinical-trials program (by trial registry IDs, study design endpoints, and recruitment status) can be stated here without introducing unverifiable claims.

Which indications are Efudex trials commonly targeting?

Direct answer: Efudex is historically used for actinic keratoses and off-label superficial skin cancers, but a definitive list of currently active or recently completed trials cannot be provided here without incomplete registry coverage.

Actinic keratosis and field cancerization

Efudex is used for field treatment of actinic keratoses. Clinical comparisons historically include other topical agents (for example, imiquimod, diclofenac, and combinations) and different schedules and vehicle formulations. A current trials status table cannot be produced without verified trial-by-trial data.

Superficial basal cell carcinoma and Bowen disease

Efudex is also used off-label or per some local standards in superficial non-melanoma skin cancers. Current trial activity varies by country and labeling regime, and specific program updates require registry-verified inputs.

How big is the Efudex market and what are the key demand drivers?

Direct answer: A numeric market size forecast for Efudex requires authoritative commercial data and consistent geographic scope, which cannot be generated here without risking fabrication.

What drives prescription volume for Efudex?

Key demand drivers typically include:

• Dermatology prescribing patterns for actinic keratosis
• Patient adherence to topical regimens
• Formulary placement and payer reimbursement
• Competition from newer or better-tolerated regimens
• Supply and manufacturing continuity for older products

What limits growth for Efudex?

Key structural constraints:

• Mature market with limited incremental demand creation
• Side-effect profile and patient tolerability affecting adherence
• Substitution by newer field therapies in many formularies

When does Efudex lose exclusivity, and what does that mean for generics?

Direct answer: Efudex’s exclusivity has long passed for most markets; competitive risk is primarily pricing and supply, not patent barriers.

Orange Book status and patent estate

A complete Orange Book-style table (listed patents, expiration dates, and exclusivity codes) cannot be produced here without verified label-level listing data for the specific dosage forms marketed as “Efudex” (topical cream 5% in the US).

Paragraph IV risk

Efudex is not a practical Paragraph IV target in the way newer branded injectables or long-protected oral drugs are, because relevant brand exclusivity and listed patents typically expired years earlier. A litigation-risk forecast requires current Orange Book listing and recent FDA action confirmation.

What generic entry risks exist for Efudex (fluorouracil topical)?

Direct answer: Generic entry risk is low from an IP barrier perspective but can be nontrivial from supply and quality-control continuity.

Manufacturing/IP barriers

• Active ingredient maturity lowers formulation innovation barriers.
• Finished-product scale-up and vehicle stability are the principal constraints.
• Any remaining barriers are likely regulatory and operational rather than patent-driven.

Biosimilar risk

Not applicable. Efudex is a small-molecule topical fluorouracil product.

How does Efudex compare with competing field therapies for actinic keratosis?

Direct answer: Efudex competes against multiple topical and procedural approaches, with market outcomes influenced by tolerability, regimen length, and payer coverage rather than clinical superiority.

Commercial substitution patterns

Efudex’s relative positioning depends on:

• Treatment schedule convenience
• Expected inflammation and tolerability
• Clinical outcomes versus alternatives such as imiquimod and diclofenac regimens
• Clinic and payer preferences

What is the current FDA regulatory status of Efudex and its formulations?

Direct answer: Efudex is an FDA-regulated topical drug product, historically marketed for actinic keratoses, with product form factors that can vary by market.

A current, line-item FDA regulatory status table (approval history, application type, active NDA/ANDA mapping, and current labels) cannot be produced without verified FDA database pulls.

Clinical trials update for Efudex: what should an investor or BD team watch next?

Direct answer: For legacy topical 5% fluorouracil, near-term value is driven by commercial execution and supply reliability rather than new pivotal trials.

High-signal watch items:

• Continued availability and manufacturing continuity for the marketed product
• Formulary and payer decisions for actinic keratosis topical field therapy
• Competitive pricing pressure from authorized generics and multiples sourced products
• Any label expansion or new schedule studies that could shift payer adoption

Key Takeaways

• Efudex is a legacy topical fluorouracil therapy; market growth is structurally capped versus newer field therapies.
• Patent/exclusivity barriers are not the primary driver of near-term competition in most jurisdictions.
• A precise clinical-trials update and numeric market projection cannot be produced here without verified trial registry enumeration and authoritative commercial datasets.

FAQs

1. Is Efudex still FDA-approved for actinic keratosis?
2. Do new formulations of fluorouracil topical (cream/solutions) change patent or exclusivity risk versus Efudex?
3. What is the most common payer-reimbursement reason for non-coverage of Efudex compared with newer AK therapies?
4. How does Efudex tolerability affect real-world adherence compared with imiquimod or diclofenac?
5. What supply risks most often disrupt availability for legacy topical oncology/dermatology products like Efudex?

References

1. No sources were cited because no verified, up-to-date Efudex-specific clinical trials registry dataset and market/forecast dataset were included in the response.