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Fluorouracil (5FU)
Fluorouracil (5FU) is a Antimetabolite Small molecule drug developed by University of Chicago. It is currently in Phase 3 development for Colorectal cancer, Breast cancer, Gastric cancer. Also known as: Tolak, Fluoroplex, Efudex.
Fluorouracil is a pyrimidine analog that inhibits thymidylate synthase and gets incorporated into RNA and DNA, disrupting nucleotide synthesis and causing cancer cell death.
Fluorouracil is a pyrimidine analog that inhibits thymidylate synthase and gets incorporated into RNA and DNA, disrupting nucleotide synthesis and causing cancer cell death. Used for Colorectal cancer, Breast cancer, Gastric cancer.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Fluorouracil (5FU) |
|---|---|
| Also known as | Tolak, Fluoroplex, Efudex |
| Sponsor | University of Chicago |
| Drug class | Antimetabolite |
| Target | Thymidylate synthase |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
5-FU is converted intracellularly to active metabolites that inhibit thymidylate synthase, blocking dTMP synthesis and DNA replication. It also incorporates into RNA, disrupting protein synthesis. These dual mechanisms make it effective against rapidly dividing cancer cells.
Approved indications
- Colorectal cancer
- Breast cancer
- Gastric cancer
- Pancreatic cancer
- Head and neck cancer
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Mucositis
- Diarrhea
- Nausea and vomiting
- Hand-foot syndrome
- Alopecia
Key clinical trials
- Therapy Adapted for High Risk and Low Risk HIV-Associated Anal Cancer (PHASE2)
- A Study to Evaluate the Safety and Efficacy of Two Dose Levels of ONO-4578 With Opdivo®, in Combination With mFOLFOX6 and Bevacizumab Versus Standard of Care in Participants With Non-MSI-H/dMMR, PD-L1 Positive Advanced Colorectal Cancer (PHASE2)
- Neoadjuvant mFolfirinox With or Without Preoperative Concomitant Chemoradiotherapy in Patients With Borderline Resectable Pancreatic Carcinoma (PANDAS-PRODIGE 44) (PHASE2)
- Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial (PHASE2)
- Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer (PHASE3)
- Individual Response to Hyperthermic Intraperitoneal Chemotherapy (HIPEC) Treatment of Peritoneal Carcinomatosis From Peritoneal Mesothelioma or Atypical Mesothelial Proliferation or From Ovarian, Colorectal, or Appendiceal Histologies (PHASE1)
- Studying Chemotherapy With or Without Panitumumab for Unresectable, Locally Advanced, or Metastatic Pancreatic Cancer Without KRAS Mutations (PHASE3)
- Testing the Addition of the Immunotherapy Drug, Pembrolizumab, to Radiation Therapy Compared to the Usual Chemotherapy Treatment During Radiation Therapy for Bladder Cancer, PARRC Trial (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Fluorouracil (5FU) CI brief — competitive landscape report
- Fluorouracil (5FU) updates RSS · CI watch RSS
- University of Chicago portfolio CI
Frequently asked questions about Fluorouracil (5FU)
What is Fluorouracil (5FU)?
How does Fluorouracil (5FU) work?
What is Fluorouracil (5FU) used for?
Who makes Fluorouracil (5FU)?
Is Fluorouracil (5FU) also known as anything else?
What drug class is Fluorouracil (5FU) in?
What development phase is Fluorouracil (5FU) in?
What are the side effects of Fluorouracil (5FU)?
What does Fluorouracil (5FU) target?
Related
- Drug class: All Antimetabolite drugs
- Target: All drugs targeting Thymidylate synthase
- Manufacturer: University of Chicago — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Colorectal cancer
- Indication: Drugs for Breast cancer
- Indication: Drugs for Gastric cancer
- Also known as: Tolak, Fluoroplex, Efudex
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing