CLINICAL TRIALS PROFILE FOR FLUOROURACIL

505(b)(2) Clinical Trials for fluorouracil

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	NCT01489865 ↗	ABT-888 With Modified FOLFOX6 in Patients With Metastatic Pancreatic Cancer	Unknown status	Abbott	Phase 1/Phase 2	2011-02-01	People are being asked to participate in this study who have metastatic pancreatic cancer (cancer that has spread to other parts of the body). The purpose of this study is to test the efficacy (effectiveness) of a new combination of drugs, ABT-888 and mFOLFOX-6 (modified 5-Fluorouracil and Oxaliplatin) for patients with metastatic pancreatic cancer. ABT-888 inhibits an enzyme called "PARP" which helps to fix damaged DNA. By inhibiting this enzyme, ABT-888 prevents cancer cells from repairing the damage caused by the mFOLFOX-6, and will hopefully increase the killing of cancer cells, thus decreasing the tumors in your body.
New Combination	NCT01489865 ↗	ABT-888 With Modified FOLFOX6 in Patients With Metastatic Pancreatic Cancer	Unknown status	Georgetown University	Phase 1/Phase 2	2011-02-01	People are being asked to participate in this study who have metastatic pancreatic cancer (cancer that has spread to other parts of the body). The purpose of this study is to test the efficacy (effectiveness) of a new combination of drugs, ABT-888 and mFOLFOX-6 (modified 5-Fluorouracil and Oxaliplatin) for patients with metastatic pancreatic cancer. ABT-888 inhibits an enzyme called "PARP" which helps to fix damaged DNA. By inhibiting this enzyme, ABT-888 prevents cancer cells from repairing the damage caused by the mFOLFOX-6, and will hopefully increase the killing of cancer cells, thus decreasing the tumors in your body.
New Combination	NCT01522989 ↗	PD-0332991, 5-FU, and Oxaliplatin for Advanced Solid Tumor Malignancies	Unknown status	Pfizer	Phase 1	2011-12-01	This study is for patients with advanced solid tumor malignancies (cancer that has spread to other parts of the body). The purpose of this study is to test the safety and effectiveness of a new combination of drugs, PD-0332991 and 5-Fluorouracil and Oxaliplatin for patients with advanced solid tumor malignancies . PD-0332991 stops cells from dividing by blocking an enzyme called cyclin-dependent kinase (CDK), which cancer cells need to grow and divide. By inhibiting this enzyme, PD-0332991 prevent cancer cells from growing and dividing, while the 5-Fluorouracil and Oxaliplatin damage the cells, hopefully increasing the killing of cancer cells, thus decreasing the tumors in the body. PD-0332991 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration for use in colorectal cancer. It is given as a pill which is taken once a day for one week followed by one week off. 5-Fluorouracil and Oxaliplatin are administered as an infusion into a vein once every 2 weeks and are approved for and used as chemotherapy for several cancers.
New Combination	NCT01522989 ↗	PD-0332991, 5-FU, and Oxaliplatin for Advanced Solid Tumor Malignancies	Unknown status	Georgetown University	Phase 1	2011-12-01	This study is for patients with advanced solid tumor malignancies (cancer that has spread to other parts of the body). The purpose of this study is to test the safety and effectiveness of a new combination of drugs, PD-0332991 and 5-Fluorouracil and Oxaliplatin for patients with advanced solid tumor malignancies . PD-0332991 stops cells from dividing by blocking an enzyme called cyclin-dependent kinase (CDK), which cancer cells need to grow and divide. By inhibiting this enzyme, PD-0332991 prevent cancer cells from growing and dividing, while the 5-Fluorouracil and Oxaliplatin damage the cells, hopefully increasing the killing of cancer cells, thus decreasing the tumors in the body. PD-0332991 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration for use in colorectal cancer. It is given as a pill which is taken once a day for one week followed by one week off. 5-Fluorouracil and Oxaliplatin are administered as an infusion into a vein once every 2 weeks and are approved for and used as chemotherapy for several cancers.

All Clinical Trials for fluorouracil

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000122 ↗	Fluorouracil Filtering Surgery Study (FFSS)	Completed	National Eye Institute (NEI)	Phase 3	1985-09-01	To determine whether postoperative subconjunctival injections of 5-fluorouracil (5-FU) increase the success rate of filtering surgery in patients at high risk for failure after standard glaucoma filtering surgery.
NCT00000758 ↗	A Phase III Randomized Trial of Topical Vaginal Fluorouracil (5-Fluorouracil, 5-FU) Maintenance Therapy Versus Observation After Standard Treatment for High-Grade Cervical Dysplasia in HIV-Infected Women	Completed	Hoffmann-La Roche	Phase 3	1969-12-31	To determine the efficacy and safety of intravaginal fluorouracil administered as prophylaxis in HIV-infected women who have received standard ablative therapy (surgery) for high-grade cervical dysplasia (pre-cancer of the cervix; cervical intraepithelial neoplasia). To correlate time to recurrence of cervical dysplasia with T-cell function. Women with HIV infection are at greater risk for cervical dysplasia. Because of the likelihood that untreated or recurrent cervical dysplasia may progress to invasive cancer, there is an urgent need to develop appropriate therapies.
NCT00000758 ↗	A Phase III Randomized Trial of Topical Vaginal Fluorouracil (5-Fluorouracil, 5-FU) Maintenance Therapy Versus Observation After Standard Treatment for High-Grade Cervical Dysplasia in HIV-Infected Women	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1969-12-31	To determine the efficacy and safety of intravaginal fluorouracil administered as prophylaxis in HIV-infected women who have received standard ablative therapy (surgery) for high-grade cervical dysplasia (pre-cancer of the cervix; cervical intraepithelial neoplasia). To correlate time to recurrence of cervical dysplasia with T-cell function. Women with HIV infection are at greater risk for cervical dysplasia. Because of the likelihood that untreated or recurrent cervical dysplasia may progress to invasive cancer, there is an urgent need to develop appropriate therapies.
NCT00001165 ↗	Combination Chemotherapy in Patients With Zollinger-Ellison Syndrome and Tumors of the Pancreas	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2	1978-09-01	Patients with Zollinger-Ellison Syndrome suffer from ulcers of the upper gastrointestinal tract, higher than normal levels of gastric acid, and tumors of the pancreas known as non-beta islet cell tumors. Prior to the use of drugs to cure the ulcers, patients typically died due to severe ulcers. Because of such effective drugs to treat the ulcers it is more common to see patients dying due to the pancreatic tumors. The study will observe patients suffering from Zollinger-Ellison Syndrome and non-beta islet cell tumors and determine the effectiveness of combined chemotherapy with streptozotocin, 5-fluorouracil, and doxorubicin.

Clinical Trial Conditions for fluorouracil

Condition Name

Condition Name for fluorouracil
Intervention	Trials
Colorectal Cancer	297
Breast Cancer	124
Gastric Cancer	113
Pancreatic Cancer	111

Condition MeSH

Condition MeSH for fluorouracil
Intervention	Trials
Colorectal Neoplasms	556
Adenocarcinoma	270
Carcinoma	250
Pancreatic Neoplasms	209

Clinical Trial Locations for fluorouracil

Trials by Country

Trials by Country for fluorouracil
Location	Trials
China	653
United States	5,983
Japan	397
Canada	380
Italy	342

Trials by US State

Trials by US State for fluorouracil
Location	Trials
California	284
New York	273
Texas	239
Illinois	226
Florida	211

Clinical Trial Progress for fluorouracil

Clinical Trial Phase

Clinical Trial Phase for fluorouracil
Clinical Trial Phase	Trials
PHASE4	6
PHASE3	36
PHASE2	104

Clinical Trial Status

Clinical Trial Status for fluorouracil
Clinical Trial Phase	Trials
Completed	858
RECRUITING	390
Unknown status	238

Clinical Trial Sponsors for fluorouracil

Sponsor Name

Sponsor Name for fluorouracil
Sponsor	Trials
National Cancer Institute (NCI)	386
Sun Yat-sen University	70
Sanofi	66

Sponsor Type

Sponsor Type for fluorouracil
Sponsor	Trials
Other	2405
Industry	783
NIH	395

Fluorouracil Clinical Trials Update, Market Analysis, and 2030+ Revenue Projections (5-FU)

Last updated: July 27, 2026

Fluorouracil (5-fluorouracil, 5-FU) is an off-patent, widely used chemotherapy with decades of clinical use, limiting the value of “new entrant” patent estates for future market share. Near-term growth is driven by (1) substitution dynamics between 5-FU-based regimens and newer standards of care, (2) regimen intensity shifts across metastatic colorectal and head and neck cancers, and (3) biosupply stability and manufacturing economics rather than proprietary product launches. Clinical trial activity remains concentrated in combination regimens, delivery systems, and biomarker-enriched strategies rather than first-in-class monotherapy development.

What clinical trials are currently recruiting or ongoing for fluorouracil (5-FU)?

Featured snippet: Active clinical efforts focus on 5-FU in combinations (with immunotherapy, targeted agents, and other cytotoxics), optimized dosing schedules, and delivery approaches that aim to improve therapeutic index.

What trial types dominate 5-FU studies?

• Combination regimens
  • With immune checkpoint inhibitors in gastrointestinal oncology and head and neck squamous cell carcinoma.
  • With targeted agents used in metastatic colorectal cancer, including VEGF-pathway agents and EGFR-pathway agents depending on RAS/BRAF status.
• Schedule and regimen optimization
  • Comparisons of bolus vs infusional approaches.
  • Intensification or de-escalation in neoadjuvant and adjuvant settings where 5-FU is backbone therapy.
• Delivery and formulation
  • Attempts to improve exposure-control and reduce mucositis or cardiotoxicity risk.
  • Liposomal or sustained-release concepts in exploratory phases.
• Biomarker selection
  • Studies using DPD (dihydropyrimidine dehydrogenase) testing or related pharmacogenomic approaches to manage toxicity.
  • Trials examining response predictors tied to tumor genotype and microenvironment.

Which cancer types are most represented?

• Metastatic colorectal cancer (mCRC)
• Locally advanced and recurrent head and neck squamous cell carcinoma (HNSCC)
• Gastroesophageal cancers (including gastric and gastroesophageal junction settings)
• Pancreatic cancer combinations (smaller but recurring presence in phase II/III program portfolios)
• Other solid tumors where 5-FU remains standard-of-care in certain lines

Trial timeline outlook

• 2026-2028: Most registrable value is expected from readouts of combination strategies and schedule optimization rather than monotherapy superiority.
• 2028-2031: Likely continuation of biomarker-enriched cohorts and sequencing strategies that affect how often 5-FU regimens remain preferred over alternatives.

How big is the fluorouracil (5-FU) market and what drives demand?

Featured snippet: Global demand tracks largely with the incidence and treated prevalence of colorectal and head and neck cancers, adjusted for regimen preference and drug-cost dynamics across health systems.

Core demand drivers

1. 5-FU backbone role
   • Used as foundation in multiple chemo regimens, including those paired with leucovorin and oxaliplatin or irinotecan in colorectal cancer.
2. Hospital/infusion practice patterns
   • Infusional and schedule preferences vary by region and institution, affecting utilization but not changing the underlying dependence on 5-FU.
3. Wastage, procurement, and supply
   • Because 5-FU is commodity-like, pricing pressure is tied to manufacturing capacity and supply continuity.
4. Toxicity management
   • DPD testing and dose adjustment policies reduce severe toxicity and may stabilize use in medically fit patients.

Supply and manufacturing economics

5-FU is vulnerable to supply shocks typical for mature sterile injectables. Market outcomes are often more sensitive to availability, drug master file access, and sterile fill-finish capacity than to clinical differentiation.

When does fluorouracil lose exclusivity or face generic substitution pressure?

Featured snippet: Fluorouracil is not protected by meaningful commercial exclusivity in most jurisdictions; market dynamics are primarily generic and supply-driven.

Exclusivity and patent landscape implications

• Drug substance and many old formulation protections are expired.
• Competitive pressure is structurally constant due to low switching costs and widespread generic availability.
• Any incremental differentiation typically sits in:
  • specific fixed-dose combinations (where applicable),
  • sterile manufacturing improvements,
  • and niche delivery concepts that may still be under development.

What does this mean for market share?

• Share is driven by:
  • contracting and tenders
  • supply reliability
  • unit cost
  • line-item pharmacy formularies
• “Clinical wins” in trials do not translate cleanly to pricing power unless they support a distinct labeled regimen or delivery product.

How do fluorouracil regimens compare with capecitabine and newer agents?

Featured snippet: 5-FU competes most directly with oral prodrugs like capecitabine and indirectly with regimen alternatives that aim to improve overall survival or reduce toxicity.

Key comparative axes

• Route and administration
  • 5-FU (infusional/bolus) is typically administered in infusion settings.
  • Capecitabine is oral and often chosen when patient convenience and adherence support it.
• Toxicity profiles
  • Both are fluoropyrimidines; toxicity management overlaps (mucositis, diarrhea, neutropenia).
  • Delivery route changes practical toxicity and monitoring intensity.
• Line of therapy and combination standards
  • Newer biologics and small-molecule targeted therapies shape how often a fluoropyrimidine backbone is used, not whether it is used in general.

Practical market implication

• A shift toward oral therapy increases unit substitution away from injectable 5-FU unless infusion services are structured to maintain 5-FU selection.
• Conversely, persistence of infusional regimens in certain settings preserves 5-FU demand.

What patent and litigation risks affect fluorouracil commercialization?

Featured snippet: Commercial risk is low from a traditional exclusivity standpoint; the primary risks are supply chain, regulatory enforcement for sterile manufacturing, and any localized patent barriers to specific combinations or formulations.

Patent estate reality for 5-FU

• Mature drug with expired core protections.
• Patent litigation impact is typically concentrated on:
  • fixed-dose combinations,
  • method-of-use claims tied to specific dosing regimens,
  • and formulation/manufacturing claims that could still exist in limited scopes.

What matters in practice

• Regulatory compliance and sterile manufacturing inspections.
• ANDA approval ecosystems that govern access and supply.

What is the FDA regulatory status of fluorouracil and how is it labeled?

Featured snippet: Fluorouracil is FDA-approved for multiple oncology indications as an injectable chemotherapy, typically supplied as sterile solutions and used across standardized chemo regimens.

Typical labeled positioning (high level)

• Indications span several solid tumors where fluorouracil is standard in combination regimens.
• FDA labeling is used mainly to ensure:
  • appropriate dosing references,
  • contraindications related to toxicity management,
  • and compatible administration schedules with supportive care.

Biosimilar or biologic pathway relevance

• 5-FU is not a biologic, so “biosimilar” frameworks do not apply.

What formulations of fluorouracil are protected by patents, and which are generic-dominated?

Featured snippet: Most sterile injectable presentations are generic-dominated due to expired protections; any remaining formulation barriers usually sit in narrow combinations or specific manufacturing processes.

Common presentation categories (market-relevant)

• Sterile injectable solutions
• Combination products (where historically present)
• Infusional administration formats
• Any niche delivery systems remain mostly investigational unless commercialized in a distinct labeled product.

Practical implication for business

• R&D ROI is typically highest in:
  • improving patient experience or workflow (stability, packaging, compatibility),
  • or reducing toxicity through dosing strategies tied to pharmacogenomics.

What generic entry risks exist for fluorouracil in 2026-2030?

Featured snippet: Entry risk is driven more by sterile manufacturing and regulatory compliance than by IP. Economics are constrained by low margin pricing and tender-driven procurement.

Barriers to entry that still matter

• Fill-finish capacity and sterile compliance history.
• Batch consistency and impurity profiles.
• ANDA submission quality and facility readiness.
• Supply chain constraints for starting materials.

What could disrupt supply or pricing

• Rapid capacity expansions by generics can pressure prices.
• Plant shutdowns or inspection outcomes can produce short-term scarcity and price spikes.

How many patents cover fluorouracil (5-FU) across major jurisdictions?

Featured snippet: The meaningful count for enforceable commercial exclusivity is low for the active ingredient; the larger “patent footprint” includes expired substance/formulation claims and narrow, potentially actionable combinations.

Jurisdictional pattern

• US and Europe: Older drug substance and early formulations are largely expired; any remaining actionable claims are typically tied to niche aspects (specific regimen claims or combinations).
• Japan and other markets: Similar end-state where mature drug protections have lapsed.

Business takeaway

• For market entry or competition planning, operational/regulatory and procurement dynamics outweigh the active IP barrier risk for standard 5-FU injectables.

Which companies are major players in fluorouracil supply, and how does that affect access and pricing?

Featured snippet: The supply base is dominated by established generic sterile injectables manufacturers and distributors. Competitive intensity typically yields tender-driven price compression.

How to think about “who wins”

• Contracting performance and bid competitiveness
• Ability to maintain continuous supply
• Track record in sterile inspections
• Distribution network coverage and tender alignment

Commercial risk profile

• Lower differentiation.
• Higher sensitivity to manufacturing disruptions and procurement cycles.

Market projection for fluorouracil (5-FU): 2026-2030 revenue outlook

Featured snippet: Revenue growth is likely modest and driven by volume stability and incremental regimen retention rather than brand-like pricing power. Pricing pressure remains structurally downward.

Projection logic (directional)

• Volume: tied to cancer incidence and treatment rates for fluoropyrimidine-dependent regimens.
• Price: tends to decline with generic competition, with periodic spikes from supply constraints.
• Mix: shifts between bolus and infusional schedules, and between injectable 5-FU and oral capecitabine, affect unit volumes.

Baseline scenario (directional)

• 2026-2027: Stable to slight growth in utilization with ongoing pricing compression and occasional supply-driven pricing events.
• 2028-2030: Flat-to-low growth as standard-of-care stabilizes around existing fluoropyrimidine backbones and newer agents further segment lines of therapy.

Upside/downside triggers

• Upside:
  • regimen expansion in additional indications where 5-FU remains a backbone,
  • sustained infusion-center adoption in regions where oral alternatives are less preferred.
• Downside:
  • further substitution to oral capecitabine,
  • shifts to non-fluoropyrimidine regimens in some settings,
  • repeated sterile supply disruptions causing non-availability and missed treatment schedules.

How do clinical trial results likely influence fluorouracil use through 2030?

Featured snippet: Trial outcomes that improve tolerability and regimen sequencing are most likely to sustain fluorouracil backbone usage, even without survival breakthroughs.

Most plausible value from trials

• Reduced toxicity enabling full-dose delivery
• Biomarker-guided dosing and improved safety
• Enhanced response rates when combined with contemporary systemic therapies
• Better operational fit in combination regimens (administration schedule, infusion duration compatibility)

What would change the market meaningfully

• A demonstration that a fluoropyrimidine-free regimen becomes dominant in a large fluoropyrimidine backbone segment with clear clinical superiority and manageable toxicity.
• Regulatory or guideline shifts that move fluorouracil away from backbone status in high-volume indications.

Key Takeaways

• 5-FU clinical development is dominated by combination regimens, schedule optimization, and biomarker-driven toxicity management rather than new monotherapy differentiation.
• Market growth is structurally limited by generic competition and low pricing power; revenue outcomes depend on volume stability, supply continuity, and tender dynamics.
• The most meaningful “future moat” is operational: sterile manufacturing reliability, contracting execution, and formulation stability that reduce shortages and treatment interruptions.
• From 2026-2030, the base case is modest growth with downward pressure on net price; clinical trial readouts mainly affect mix and retention of 5-FU as a backbone regimen.

FAQs

1. Are there active phase 3 trials using fluorouracil with immunotherapy in colorectal cancer?
2. How does DPD testing change fluorouracil dosing and reduce toxicity in clinical practice?
3. What are the main differences in clinical outcomes between infusional 5-FU and bolus regimens?
4. Does fluorouracil face supply shortages that can drive pricing volatility?
5. How does capecitabine substitution affect long-term demand for injectable fluorouracil?

References

1. ClinicalTrials.gov. (n.d.). Fluorouracil trials. https://clinicaltrials.gov/
2. FDA. (n.d.). Drug labels for fluorouracil (5-FU) injectable products. https://www.accessdata.fda.gov/
3. NCCN Guidelines. (n.d.). Colon cancer and head and neck cancer treatment recommendations. https://www.nccn.org/