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Last update 19 Mar 2026

Fluorouracil

Last update 19 Mar 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

5-fluoro-1H-pyrimidine-2,4-dione, 5-Fluoropyrimidine-2,4-dione, 5-Fluorouracil

+ [38]

Target

TYMS

Action

inhibitors

Mechanism

TYMS inhibitors(Thymidylate synthase inhibitors)

Therapeutic Areas

NeoplasmsDigestive System DisordersEndocrinology and Metabolic Disease+ [10]

Active Indication

Pancreatic adenocarcinomastomach adenocarcinomaEsophageal Carcinoma+ [99]

Inactive Indication

Acinar Cell Carcinomaadenocarcinoma of biliary tractAdenocarcinoma, metastatic+ [117]

Originator Organization

Spectrum Pharmaceuticals, Inc.

Active Organization

National Cancer Institute

Northwestern UniversityECOG-ACRIN Medical Research Foundation, Inc.

+ [26]

Inactive Organization

Sanofi

Roswell Park Comprehensive Cancer Center

OSI Pharmaceuticals, Inc.

+ [48]

License Organization

Dr. Reddy's Laboratories Ltd.

Hill Dermaceuticals, Inc.

Extrovis AG

+ [1]

Drug Highest PhaseApproved

First Approval Date

United States (25 Apr 1962),

Breast CancerColorectal CancerPancreatic Cancer+ [1]

RegulationOrphan Drug (United States)

Structure/Sequence

Molecular FormulaC4H3FN2O2

InChIKeyGHASVSINZRGABV-UHFFFAOYSA-N

CAS Registry51-21-8

2,776

Clinical Trials associated with Fluorouracil

NCT07363408

/ Not yet recruitingPhase 1IIT

A Phase I Clinical Trial of Ivonescimab and ADG126, Alone, and in Combination With 5-FU/LV or FOLFIRI in MSS Advanced/Metastatic Colorectal Cancer

This phase I trial studies the safety, side effects, and best dose of ADG126, in combination with ivonescimab alone, in combination with ivonescimab, leucovorin, and fluorouracil, or in combination with ivonescimab and leucovorin, fluorouracil, and irinotecan (FOLFIRI regimen) in treating patients with microsatellite stable (MSS) colorectal cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or has spread from where it first started (primary site) to other places in the body (metastatic). Immunotherapy with monoclonal antibodies, such as ivonescimab and ADG126, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Ivonescimab may also stop or slow the cancer by blocking the growth of new blood vessels necessary for tumor growth. Leucovorin calcium is a type of drug called a folic acid analog, which means it is similar to the vitamin folic acid. It is used in combination with certain chemotherapy drugs to enhance their ability to kill tumor cells or to lessen their harmful side effects. Fluorouracil is a type of chemotherapy called an antimetabolite, which is a drug that mimics a natural chemical and prevents its use in cells. It interferes with the production of a key component of deoxyribonucleic acid (DNA), which prevents the DNA from copying itself. This causes tumor cells and other rapidly dividing cells to die. Fluorouracil also gets incorporated into ribonucleic acid (RNA) and DNA, disrupting critical cell functions. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Giving ADG126 with ivonescimab, with or without leucovorin and fluorouracil or FOLFIRI regimen, may be safe in treating patients with MSS advanced/metastatic colorectal cancer.

Start Date21 Jul 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT07437287

/ Not yet recruitingPhase 2/3IIT

Countering Immunotherapy Resistance With Novel Combinations in Advanced Biliary Tract Cancers

The goal of this clinical trial is to etablish whether adding Mitazalimab to standard chemotherapy is more effective than standard chemotherapy alone in people with advanced bile duct cancer. It will also learn about the safety of Mitazalimab.
The main questions it aims to answer are:
* Does the addition of Mitazalimab enhance efficacy?
* What medical problems do participants have when taking Mitazalimab + mFOLFOX?
Participants will:
* Take drug mFOLFOX every two weeks until disease progression or mFOLFOX every two weeks plus mitazalimab in addition to mFOLFOX, with a first injection 7 days before the first mFOLFOX chemotherapy and then 3 days after the start of each mFOLFOX cycle.
* Visit the clinic once every 2 weeks for checkups and tests
* Have a radiological assessment every 8 weeks during treatment. After stopping treatment, participants will be monitored at the hospital every 8 weeks if no progression is observed, or every 12 weeks after disease progression.

Start Date01 Jul 2026

Sponsor / Collaborator

UNICANCER

[+1]

NCT07271355

/ Not yet recruitingPhase 3IIT

Investigation of Mitomycin C PIPAC - FOLFIRI Combination for Unresectable Appendiceal or Colorectal Peritoneal Metastases Treatment (IMPACT): A Multicenter, Randomized, Open-Label, Phase 3 Trial

This phase III trial studies how well pressurized intraperitoneal aerosolized chemotherapy (PIPAC) with mitomycin works versus (vs) standard chemotherapy (leucovorin calcium, fluorouracil, and irinotecan hydrochloride [FOLFIRI regimen] plus bevacizumab) in treating patients with appendix or colorectal cancer that cannot be removed by surgery (unresectable) and has spread from where it first started (primary site) to the abdominal cavity (peritoneal metastases). PIPAC is a new therapeutic approach that is minimally invasive, does not require surgery (laparotomy), and can be frequently repeated. Chemotherapy is delivered as a pressurized mist directly inside the abdominal cavity (peritoneum) during a minimally invasive surgery called a laparoscopy. The pressure helps the chemotherapy absorb into the cancer tissue and spread more evenly. Mitomycin is an antibiotic used as a chemotherapy drug. It stops or slows the growth of cancer cells and other rapidly growing cells by damaging their deoxyribonucleic acid (DNA). Standard chemotherapy drugs, such as those in the FOLFIRI regimen, are given via infusion into a vein (intravenously), and work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Another standard intravenous drug, bevacizumab, is in a class of medications called antiangiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to tumor. This may slow the growth and spread of tumor. Giving mitomycin via PIPAC in combination with the standard FOLFIRI regimen, with or without bevacizumab, may work better than standard FOLFIRI plus bevacizumab alone in treating patients with unresectable appendix or colorectal cancer with peritoneal metastases.

Start Date01 Jul 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

100 Clinical Results associated with Fluorouracil

100 Translational Medicine associated with Fluorouracil

100 Patents (Medical) associated with Fluorouracil

56,679

Literatures (Medical) associated with Fluorouracil

31 Dec 2026·Gut Microbes

Exploring the gut microbiome and metabolomic interactions of antimetabolite drugs to optimize therapy

Review

Author: Zhao, Libo ; Chen, Jingyang ; Wang, Yanan ; Xu, Lei ; Li, Xiaona

Antimetabolite drugs are cornerstones in treating various cancers and autoimmune diseases; however, their clinical utility is often hampered by systemic toxicity caused by drug-induced gut microbiota dysbiosis. Predicting patient responses remains a significant challenge. Several studies have highlighted the influence of gut microbiota on antimetabolite treatment outcomes, revealing complex bidirectional interactions between the drugs and microbial communities. This review synthesizes the effects of common antimetabolites (including 5-fluorouracil, methotrexate, gemcitabine, capecitabine, 6-mercaptopurine, and thioguanine) on gut microbial communities and outlines a framework (pharmacokinetics, endogenous metabolite production, immune modulation, and apoptotic pathway modulation) for assessing chemotherapy-microbiota interactions. Additionally, potential microbial biomarkers for predicting treatment responses and strategies for manipulating the gut microbiota to enhance therapeutic efficacy are discussed. Therefore, advances in methodologies such as metagenomics and real-time microbial monitoring will be essential for unraveling these interactions and promoting the precise application of antimetabolite drugs.

01 Dec 2026·JOURNAL OF BIOLOGICAL PHYSICS

Difference in cell death response between mitomycin C and 5-fluorouracil treatment studied using quartz crystal microbalance combined with simultaneous monitoring of viable cells

Article

Author: Muramatsu, Hiroshi ; Long, Hao ; Sugiyama, Tomoyasu

A quartz crystal microbalance (QCM) can be used to evaluate the physical properties of cells exposed to anticancer drugs. Mass-sensing techniques on electrodes detect subtle changes in adherent cells during drug treatment, providing insights into physiological, biochemical, and morphological events from a physical perspective. Although these methods have been established in many studies using living cells, their drug responses remain associated with cell viability. This study aimed to measure QCM response by simultaneously monitoring non-viable cell images. Treatment with mitomycin C (MMC) caused the resonant frequency to shift from a decrease to an increase, followed by a delayed rise in the proportion of non-viable cells. By contrast, treatment with 5-fluorouracil (5-FU) produced minimal frequency changes, accompanied by a shorter delay before cell death was observed. The fitting data to the model equations of the cumulative log-normal distribution curve showed a clear difference in parameter values between MMC and 5-FU, indicating distinct cell death processes. These results demonstrate that QCM-based monitoring of physical properties provides complementary information on drug responses and may serve as a useful tool in anticancer drug development.

01 Dec 2026·MOLECULAR BIOLOGY REPORTS

Non-Coding RNAs and cernas: emerging modulators of drug response in colorectal cancer

Review

Author: Abkenar, Elahe Daskar ; Sadeghi, Amir ; Mojarad, Ehsan Nazemalhosseini ; Nobili, Stefania ; Shams, Elahe ; Moghani, Mona Malekzadeh ; Sina, Negin ; Ebrahimi, Sara ; Fatemi, Nayeralsadat

Colorectal cancer (CRC) is still one of the most common cancers and a leading cause of cancer morbidity worldwide. A significant issue that should be paid much attention to is its resistance to anticancer agents. Non-coding RNAs (ncRNAs), such as microRNAs (miRNAs), long non-coding RNAs (lncRNAs), and circular RNAs (circRNAs), have been identified as important regulators of drug response and drug resistance in CRC. In this review, we provide a comprehensive assessment of the studies conducted in the field of investigating the role of ncRNAs in drug resistance to prominent anticancer agents used in CRC, including 5-fluorouracil (5-FU), oxaliplatin, cetuximab, bevacizumab, and regorafenib. We focussed specifically on the miRNAs, lncRNAs, and circRNAs associated with resistance to each drug individually, even within the context of combination therapies, such as FOLFOX. We also reviewed competing endogenous RNA (ceRNAs) networks and their relationship with drug sensitivity and resistance and new therapeutic strategies to manage drug resistance in CRC and also analyzed the role of ceRNA networks in modulating drug response and its implications for new approaches to overcome drug resistance in CRC. This article also discusses findings from human clinical trials, highlighting evidence from patient studies that validate the role of targeting ceRNA and ncRNA networks in improving drug sensitivity, overcoming resistance, and enhancing therapeutic outcomes in CRC. It supports the targeting of ncRNA and ceRNA networks as potential therapies to improve treatment outcomes and drug resistance in CRC.

903

News (Medical) associated with Fluorouracil

17 Mar 2026

·www.biospace.com

What the Leucovorin Approval Signals—or Doesn’t—About the FDA’s Rare Disease Stance

FDA Commissioner Marty Makary speaks at a White House press conference on September 22, 2025. / Joyce N. Boghosian, White House Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency. The regulatory process, which relied on a literature review rather than new evidence, stands in contrast to recent rare disease rejections in which the FDA cited a need for more rigorous evidence. FDA observers have noted a disconnect between what agency officials say about the need for regulatory flexibility when considering therapeutics for rare diseases, and a string of rejections of just such candidates. UniQure, for example, has weathered not only a complete response letter (CRL) but also public criticism from FDA officials for attempting to win approval based on an externally controlled pivotal trial. RENENXBIO, Biohaven and Capricor Therapeutics have also received CRLs over the past year. Sen. Ron Johnson (R-WI) has launched an investigation into such rejections. In sharp contrast to these stories, the FDA last week touted its expansion of leucovorin—long approved to ameliorate the effects of certain cancer therapies—to treat cerebral folate deficiency (CFD) linked to a particular genetic variant, an action it said “reflects the agency’s commitment to accelerating cures and expanding treatment options including for patients with serious and unmet needs.” Notably, the expansion was initiated not by a drug company but by the FDA itself after Commissioner Marty Makary promoted leucovorin as a treatment for autism. Agency staffers mined the literature for evidence of leucovorin’s effectiveness in CFD. Unsurprisingly for a disease that has been diagnosed in fewer than 50 people worldwide, that evidence does not include a randomized, controlled trial. The apparent discrepancy in evidence standards “can’t be rationalized in a coherent sense, other than that leucovorin is a drug that is being shaped to fit RFK’s agenda around autism and it’s outside the reach of Vinay Prasad and [the Center for Biologics Evaluation and Research],” Holly Fernandez Lynch, a bioethics and law professor at the University of Pennsylvania, wrote in an email to BioSpace . “This is exactly the sort of thing that makes the public not trust FDA’s decisions, as this is not based in science, it’s based in politics.” The decision “is highly unusual—maybe unprecedented—and I think it’s good and bad,” said Paul Kim, principal at Kendall Square Policy Strategies and a former FDA counsel to the late Senator Edward Kennedy and Congressman Henry Waxman. While the regulatory approach of mining real-world evidence could benefit other rare diseases, Kim agreed there is a political story behind the expansion. A Tenuous Autism Link On Sept. 22, 2025, as part of a White House press conference in which President Donald Trump and members of his administration alleged a link between autism and Tylenol exposure in utero, FDA chief Marty Makary took the podium to deliver what he called good news. “Today, the FDA is filing a federal register notice to change the label on an exciting treatment called prescription leucovorin so that it can be available to children with autism,” he said. “We have a duty to let doctors and the public know. . . . Hundreds of thousands of kids, in my opinion, will benefit.” This came after then–Center for Drug Evaluation and Research Director George Tidmarsh had asked FDA staff in August to look into finding a way to approve leucovorin for autism, STAT News later reported. The drug, a form of folic acid, was originally sponsored by GSK and used to ameliorate the effects of cancer drugs such as methotrexate and fluorouracil. It’s been used off-label to treat CFD since 2009, an expert on the condition told CNN . GSK no longer makes the drug, but some generic suppliers do. CFD has some overlap in symptoms with autism, and a few small studies have explored leucovorin’s use in the more common condition. But according to the American Academy of Pediatrics , “Current evidence is insufficient to support prescribing leucovorin for autism in the absence of CFD.” Tidmarsh and FDA staffers ultimately compromised with a plan to submit leucovorin for approval in CFD rather than autism, STAT reported. GSK reviewed the case reports provided by the FDA and submitted the supplemental New Drug Application to update the indication, a company spokesperson told BioSpace in an email, though it does not plan to resume making or marketing the brand-name version of the drug. At the September press conference, Makary blurred the distinction between CFD and autism and discussed a possible mechanistic link between folate and autism. Policy and regulatory consultant Steven Grossman speculated that leucovorin’s approval for CFD may make the drug materially easier to access for autism as well. “[If] you’re a pediatrician and you’re under pressure from parents—and I’m sure they are—to prescribe leucovorin [for autism] because of the noises coming out of HHS,” the drug’s recent label expansion may make it easier to justify, he suggested. It would still be an off-label prescription but it would be for a drug that’s approved for a pediatric disease and not just for cancer. “While there may be more going on,” Grossman said, “the whole deal looks very much like a gigantic ‘wink-wink’ from the administration.” A Glimpse of What Could Be As Kim sees it, with the press conference and what followed, “we get this highly unsatisfactory, patient-adverse lifting of expectations and crashing of them with the announcement on CFD.” Yet for him, the episode points to what’s possible, namely that “many, many very rare diseases . . . could benefit from this approach” of mining real-world evidence. With more research funding and needed infrastructure in place, “I would be not surprised to see multiple approvals of very rare disorder therapies like leucovorin, either repurposed or novel ones, and a real renaissance,” Kim said—one that is not dependent on the financial markets or emerging company formation but rather taps into the ongoing work of academics and clinicians. As it stands, he said, the leucovorin label expansion is “like hanging a Christmas ornament high on the tree, but there’s nothing else on the tree.”

Drug Approval

16 Mar 2026

·firstwordpharma.com

AstraZeneca's Imfinzi regimen wins EU clearance in perioperative gastric cancer setting

AstraZeneca said Monday that its PD-L1 inhibitor Imfinzi (durvalumab) gained approval in the EU for the treatment of adults with resectable, early-stage and locally advanced gastric and gastroesophageal junction cancers. The clearance — for use alongside standard-of-care FLOT (fluorouracil, leucovorin, oxaliplatin and docetaxel) chemotherapy — follows a similar authorisation in the US late last year."This approval marks our third perioperative approval in Europe for an Imfinzi-based regimen," said Dave Fredrickson, EVP of AstraZeneca's oncology haematology business unit. "For patients with early gastric and gastroesophageal cancers, this immunotherapy-based regimen delivers a durable survival benefit that increases over time."The approval, following a positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) earlier this year, is based on findings from the Phase III MATTERHORN trial. In an interim analysis of the study, patients treated with the Imfinzi-based perioperative regimen showed a 29% reduction in the risk of disease progression, recurrence or death versus chemotherapy alone.Meanwhile, the final overall survival analysis showed that the Imfinzi and FLOT perioperative regimen demonstrated a statistically significant and clinically meaningful survival improvement, reducing the risk of death by 22% compared to chemotherapy alone. An estimated 69% of patients treated with the Imfinzi-based regimen were alive at three years compared with 62% in the comparator arm. AstraZeneca noted that the latest EU approval includes two cycles of Imfinzi in combination with chemotherapy before and after surgery, followed by Imfinzi monotherapy. A marketing application is currently under review in Japan for this indication.

Drug ApprovalClinical ResultImmunotherapyPhase 3

16 Mar 2026

·www.astrazeneca.com

Imfinzi approved in the EU as first and only perioperative immunotherapy for patients with early gastric and gastroesophageal cancers

AstraZeneca’s Imfinzi (durvalumab) in combination with standard-of-care FLOT chemotherapy (fluorouracil, leucovorin, oxaliplatin, and docetaxel) has been approved in the European Union (EU) for the treatment of adult patients with resectable, early-stage and locally advanced (Stages II, III, IVA) gastric and gastroesophageal junction (GEJ) cancers. The regimen includes two cycles of Imfinzi in combination with chemotherapy before and after surgery, followed by Imfinzi monotherapy. The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on the positive results from the MATTERHORN Phase III trial, which were published in The New England Journal of Medicine. Gastric cancer is the fifth leading cause of cancer death globally, with nearly one million people diagnosed each year.1 In 2024, there were roughly 15,500 drug-treated patients in the EU with early-stage and locally advanced gastric or GEJ cancer.2 Josep Tabernero, MD, PhD, head of the Medical Oncology Department at Vall d'Hebron University Hospital and director of the Vall d’Hebron Institute of Oncology (VHIO) in Barcelona, Spain, and principal investigator in the trial, said: “Despite curative-intent surgery and chemotherapy, patients with resectable gastric and gastroesophageal cancers still face high recurrence rates and an urgent need for improved long-term survival. In MATTERHORN, nearly 70 per cent of patients were still alive three years after treatment with the durvalumab-based perioperative regimen. This EU approval brings patients the first immunotherapy regimen to extend survival in this early setting and is poised to become the new standard of care.” Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said: “This approval marks our third perioperative approval in Europe for an Imfinzi-based regimen, underscoring AstraZeneca’s commitment to transforming outcomes in early-stage disease, where cure is possible. For patients with early gastric and gastroesophageal cancers, this immunotherapy-based regimen delivers a durable survival benefit that increases over time.” In a planned interim analysis, patients treated with the Imfinzi-based perioperative regimen showed a 29% reduction in the risk of disease progression, recurrence or death versus chemotherapy alone (based on an event-free survival [EFS] hazard ratio [HR] of 0.71; 95% confidence interval [CI] 0.58–0.86; p<0.001). Estimated median EFS was not yet reached for the Imfinzi arm versus 32.8 months for the comparator arm. An estimated 78.2% of patients treated with the Imfinzi-based perioperative regimen were event-free at one year, compared to 74.0% in the comparator arm; the estimated 24-month EFS rate was 67.4% versus 58.5%, respectively. In the final overall survival (OS) analysis, results showed the Imfinzi and FLOT perioperative regimen demonstrated a statistically significant and clinically meaningful survival improvement, reducing the risk of death by 22% compared to chemotherapy alone (based on a HR of 0.78; 95% CI 0.63-0.96; p=0.021). An estimated 69% of patients treated with the Imfinzi-based regimen were alive at three years compared with 62% in the comparator arm. At each subsequent prespecified OS landmark, the survival curves indicated increasing separation, signaling a greater magnitude of benefit over time for the Imfinzi-based regimen. An OS benefit was observed regardless of tumour PD-L1 status. OS results from MATTERHORN were presented in a Proffered Paper session at the European Society for Medical Oncology (ESMO) Congress 2025. The safety profile for Imfinzi and FLOT chemotherapy was consistent with the known profiles of each medicine, and the percentage of patients that completed surgery was similar compared to chemotherapy alone. Grade 3 or higher adverse events due to any cause were similar between the two arms (71.6% for Imfinzi and FLOT arm; 71.2% for comparator arm). Imfinzi and FLOT chemotherapy is approved in the US and other countries based on the MATTERHORN results. Regulatory applications are currently under review in Japan and several other countries for this indication. Notes Gastric and gastroesophageal junction cancers Gastric (stomach) cancer is the fifth most common cancer worldwide and the fifth-highest leading cause of cancer mortality.1 Nearly one million new patients were diagnosed with gastric cancer in 2022, with approximately 660,000 deaths reported globally.1 In many regions, its incidence has been increasing in patients younger than 50 years old, along with other gastrointestinal (GI) malignancies.3 GEJ cancer is a type of gastric cancer that arises from and spans the area where the oesophagus connects to the stomach.4 Disease recurrence is common in patients with resectable gastric cancer despite undergoing surgery with curative intent and treatment with neoadjuvant/adjuvant chemotherapy.5 Approximately one in four patients with gastric cancer who undergo surgery develop recurrent disease within one year, and the five-year survival rate remains poor, with less than half of patients alive at five years.5-6 MATTERHORN MATTERHORN is a randomised, double-blind, placebo-controlled, multi-centre, global Phase III trial evaluating Imfinzi as perioperative treatment for patients with resectable Stage II-IVA gastric and GEJ cancers. Perioperative therapy includes treatment before and after surgery, also known as neoadjuvant/adjuvant therapy. In the trial, 948 patients were randomised to receive a 1500mg fixed dose of Imfinzi plus FLOT chemotherapy or placebo plus FLOT chemotherapy every four weeks for two cycles prior to surgery. This was followed by Imfinzi or placebo every four weeks, for a maximum of 12 cycles after surgery (including two cycles of Imfinzi or placebo plus FLOT chemotherapy and 10 additional cycles of Imfinzi or placebo monotherapy). In the MATTERHORN trial, the primary endpoint is EFS, defined as time from randomisation until the date of one of the following events (whichever occurred first): RECIST (version 1.1, per blinded independent central review assessment) progression that precludes surgery or requires non-protocol therapy during the neoadjuvant period; RECIST progression/recurrence during the adjuvant period; non-RECIST progression that precludes surgery or requires non-protocol therapy during the neoadjuvant period or discovered during surgery; progression/recurrence confirmed by biopsy post-surgery; or death due to any cause. Key secondary endpoints include pathologic complete response rate, defined as the proportion of patients who have no detectable cancer cells in resected tumour tissue following neoadjuvant therapy, and OS. The trial enrolled participants in 176 centres in 20 countries, including in the US, Canada, Europe, South America and Asia. Imfinzi Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour's immune-evading tactics and releasing the inhibition of immune responses. In GI cancer, Imfinzi is approved in combination with chemotherapy in locally advanced or metastatic biliary tract cancer (BTC) and in combination with Imjudo (tremelimumab) in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU. In addition to its indications in GI cancers, Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy (CRT). Additionally, Imfinzi is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC, and in combination with a short course of Imjudo and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy for the treatment of extensive-stage SCLC. Perioperative Imfinzi in combination with neoadjuvant chemotherapy is approved in the US, EU, Japan and other countries for patients with muscle-invasive bladder cancer based on results from the NIAGARA Phase III trial. Additionally, in May 2025, Imfinzi added to Bacillus Calmette-Guérin induction and maintenance therapy met the primary endpoint of disease-free survival for patients with high-risk non-muscle-invasive bladder cancer in the POTOMAC Phase III trial. Imfinzi in combination with chemotherapy followed by Imfinzi monotherapy is approved as a 1st-line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in the US and EU). Imfinzi in combination with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in the EU and Japan. Since the first approval in May 2017, more than 414,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, bladder cancer, breast cancer, ovarian cancer and several GI cancers. AstraZeneca in GI cancers AstraZeneca has a broad development programme for the treatment of GI cancers across several medicines and a variety of tumour types and stages of disease. In 2022, GI cancers collectively represented approximately 5 million new cancer cases leading to approximately 3.3 million deaths.7 Within this programme, the Company is committed to improving outcomes in gastric, liver, biliary tract, oesophageal, pancreatic, and colorectal cancers. In addition to its indications in BTC and HCC, Imfinzi is being assessed in combinations, including with Imjudo, in liver, oesophageal and gastric cancers in an extensive development programme spanning early to late-stage disease across settings. Enhertu (trastuzumab deruxtecan), a HER2-directed antibody drug conjugate (ADC), is approved in the US and several other countries for HER2-positive advanced gastric cancer. Enhertu is jointly developed and commercialised by AstraZeneca and Daiichi Sankyo. Lynparza, a first-in-class PARP inhibitor, is approved in the US and several other countries for the treatment of BRCA-mutated metastatic pancreatic cancer. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. inside the US and Canada). The Company is also assessing rilvegostomig, a PD-1/TIGIT bispecific antibody, in combination with chemotherapy as an adjuvant therapy in BTC, in combination with bevacizumab with or without Imjudo as a 1st-line treatment in patients with advanced HCC, and as a 1st-line treatment in patients with HER2-negative, locally advanced unresectable or metastatic gastric and GEJ cancers. Rilvegostomig is also being evaluated in combination with Enhertu in previously untreated, HER2-expressing, locally advanced or metastatic BTC. AstraZeneca is advancing multiple modalities that provide complementary mechanisms for targeting Claudin 18.2, a promising therapeutic target in gastric cancer. These include sonesitatug vedotin, a potential first-in-class ADC licensed from KYM Biosciences Inc., currently in Phase III development; AZD5863, a novel Claudin 18.2/CD3 T-cell engager bispecific antibody licensed from Harbour Biomed in Phase I development; and AZD4360, an antibody drug conjugate, currently being evaluated in a Phase I/II trial in patients with advanced solid tumours. In early development, AstraZeneca is developing C-CAR031 / AZD7003, a Glypican 3 (GPC3) armoured CAR T, in HCC. C-CAR031 / AZD7003 is being co-developed with AbelZeta in China where it is under evaluation in an IIT. AstraZeneca in immuno-oncology (IO) AstraZeneca is a pioneer in introducing the concept of immunotherapy into dedicated clinical areas of high unmet medical need. The Company has a comprehensive and diverse IO portfolio and pipeline anchored in immunotherapies designed to overcome evasion of the anti-tumour immune response and stimulate the body’s immune system to attack tumours. AstraZeneca strives to redefine cancer care and help transform outcomes for patients with Imfinzi as a monotherapy and in combination with Imjudo as well as other novel immunotherapies and modalities. The Company is also investigating next-generation immunotherapies like bispecific antibodies and therapeutics that harness different aspects of immunity to target cancer, including cell therapy and T-cell engagers. AstraZeneca is pursuing an innovative clinical strategy to bring IO-based therapies that deliver long-term survival to new settings across a wide range of cancer types. The Company is focused on exploring novel combination approaches to help prevent treatment resistance and drive longer immune responses. With an extensive clinical programme, the Company also champions the use of IO treatment in earlier disease stages, where there is the greatest potential for cure. AstraZeneca in oncology AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients. The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience. AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death. AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Social Media @AstraZeneca. Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. References Matthew Bowden Company Secretary AstraZeneca PLC Oncology Corporate and financial

ImmunotherapyClinical ResultDrug ApprovalPhase 3Phase 2

100 Deals associated with Fluorouracil

External Link

KEGG	Wiki	ATC	Drug Bank
D00584	Fluorouracil	L01BC02	DB00544

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Pancreatic adenocarcinoma	United States	Avyxa Pharma	20 Feb 2026
Esophageal Carcinoma	Japan	Kyowa Kirin Co., Ltd.	25 Nov 2021
Intestinal Neoplasms	Japan	Kyowa Kirin Co., Ltd.	21 Sep 2018
Biliary Tract Neoplasms	Brazil	Libbs Farmacêutica Ltda.	22 Jan 2007
Bladder Cancer	Brazil	Libbs Farmacêutica Ltda.	22 Jan 2007
Colonic Cancer	Brazil	Libbs Farmacêutica Ltda.	22 Jan 2007
Gallbladder Neoplasms	Brazil	Libbs Farmacêutica Ltda.	22 Jan 2007
Hepatocellular Carcinoma	Brazil	Libbs Farmacêutica Ltda.	22 Jan 2007
Rectal Cancer	Brazil	Libbs Farmacêutica Ltda.	22 Jan 2007
Head and Neck Neoplasms	Japan	Kyowa Kirin Co., Ltd.	14 Feb 2005
Bowen's Disease	Australia	iNova Pharmaceuticals (Australia) Pty Ltd.	23 Aug 1991
Neoplasms	China	Grand Pharmaceutical (China) Co., Ltd.	01 Jan 1981
Skin Neoplasms	Japan	Kyowa Kirin Co., Ltd.	26 Aug 1972
Actinic Keratosis	United States	Extrovis AG	29 Jul 1970
Advanced gastric carcinoma	Japan	Kyowa Kirin Co., Ltd.	24 Jul 1967
Endometrial Carcinoma	Japan	Kyowa Kirin Co., Ltd.	24 Jul 1967
Liver Cancer	Japan	Kyowa Kirin Co., Ltd.	24 Jul 1967
Lung Cancer	Japan	Kyowa Kirin Co., Ltd.	24 Jul 1967
Ovarian Cancer	Japan	Kyowa Kirin Co., Ltd.	24 Jul 1967
recurrent gastric cancer	Japan	Kyowa Kirin Co., Ltd.	24 Jul 1967

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
RAS/BRAF Wild Type Colorectal Cancer	Phase 3	Italy	National Cancer Institute	12 Jun 2024
Cutaneous Squamous Cell Carcinoma	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	08 Jun 2023
Hypopharyngeal Carcinoma	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	08 Jun 2023
Squamous cell carcinoma of head and neck metastatic	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	08 Jun 2023
Squamous cell carcinoma of the hypopharynx	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	08 Jun 2023
Squamous Cell Carcinoma of the Larynx	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	08 Jun 2023
Squamous cell carcinoma of the oral cavity	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	08 Jun 2023
Advanced Gastroesophageal Junction Adenocarcinoma	Phase 3	United States	Alliance for Clinical Trials in Oncology	31 Jan 2023
Metastatic gastroesophageal adenocarcinoma	Phase 3	United States	Alliance for Clinical Trials in Oncology	31 Jan 2023
Metastatic Gastroesophageal Junction Adenocarcinoma	Phase 3	United States	Alliance for Clinical Trials in Oncology	31 Jan 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02047474 (NCT02047474 \| ###DELETE, CTgov)	Phase 2	Acinar Cell Carcinoma \| Pancreatic Ductal Adenocarcinoma	46	laboratory biomarker analysis+Leucovorin Calcium+oxaliplatin+irinotecan hydrochloride+fluorouracil	vrhyciazbt = ttbtmsbkas wibvdeehlj (tkibcfucgl, zhkbfhkiau - ksznslteml) View more	-	06 Mar 2026
NCT04094688 (SOLARIS, CTgov)	Phase 3	Adenocarcinoma of large intestine \| Metastatic Colorectal Carcinoma	455	Quality-of-Life Assessment+Leucovorin Calcium+Bevacizumab+Irinotecan Hydrochloride+oxaliplatin+Cholecalciferol+Fluorouracil (Arm I (Bevacizumab, Chemotherapy, High-dose Vitamin D3))	tudzedqanv(ssgwpzgxkv) = onrhqmjlsv bohmptkhsn (dqcdryolao, cypabfuvve - hfxjrxmejb) View more	-	03 Mar 2026
				Quality-of-Life Assessment+Leucovorin Calcium+Bevacizumab+Irinotecan Hydrochloride+oxaliplatin+Cholecalciferol+Fluorouracil (Arm II (Bevacizumab, Chemotherapy, Standard-dose Vitamin D3))	tudzedqanv(ssgwpzgxkv) = vjgxciacze bohmptkhsn (dqcdryolao, sjuisezvdb - oypwcpkoey) View more
ESGO2026	Not Applicable	Vaginal intraepithelial neoplasia	11	5-FU	cicrtzuwwf(ayfaxnewfb) = xxaywdavtt laigvkcvwz (ikuehlkuvo, 6 - 134) View more	Positive	28 Feb 2026
ASCO_GU2026	Not Applicable	Muscle Invasive Bladder Carcinoma	98	Cisplatin	osqsvrzhpk(xshgudguif) = dtawxywgdu pfacbmcpdq (guoqizchoe ) View more	Positive	26 Feb 2026
				5-FU+MMC	osqsvrzhpk(xshgudguif) = kgyuuwaecc pfacbmcpdq (guoqizchoe ) View more
NCT05567835 (TOGAR, CTgov)	Phase 2	stomach adenocarcinoma \| Gastroesophageal junction adenocarcinoma	3	GSCF+leucovorin+Docetaxel+oxaliplatin+fluorouracil (Arm A: FLOT-TNT ( Investigational Arm))	sfolaqjarm(dsqrmyoxse) = ndhsluzygb wogbjdtodt (jgcyjcmtag, lgqqmilcdu - hncpblhvks) View more	-	23 Feb 2026
				GSCF+leucovorin+Docetaxel+oxaliplatin+fluorouracil (Arm B: FLOT-POP ( Standard Arm))	sfolaqjarm(dsqrmyoxse) = enmwwetsld wogbjdtodt (jgcyjcmtag, usphclqnzg - oealroylqv) View more
NCT03785873 (BilT-03 \| BilT03, CTgov)	Phase 1/2	Advanced biliary tract cancer	34	Nivolumab+leucovorin+Nanoliposomal-Irinotecan+5-fluorouracil	kndlvyipss = jslleujfxx hjpwpafowg (fsmaimykio, lvxtyallyo - vsjehgexoh) View more	-	13 Feb 2026
NCT03693807 (CTgov)	Phase 2	Colorectal Liver Metastases \| Colorectal Cancer \| Bile Duct Neoplasms	35	Cetuximab+Gemcitabine+Floxuridine (FUDR)+Oxaliplatin+Irinotecan (CPT-11)+Panitumumab+Fluorouracil (Group 1: Unresectable Liver Metastases From Colorectal Cancer)	pslydhwmbs(ectybibliv) = becyhfluhm gbzwqjaqtz (rwmfmzetuh, dyfldbtito - yfqthhqzdx) View more	-	06 Feb 2026
				Cetuximab+Gemcitabine+Floxuridine (FUDR)+Oxaliplatin+Irinotecan (CPT-11)+Panitumumab+Fluorouracil (Group 2: Resectable Liver Metastases From Colorectal Cancer)	pslydhwmbs(ectybibliv) = cbrxkgvcbt gbzwqjaqtz (rwmfmzetuh, rwsfoikkyp - xyjcjdbrkg) View more
NCT05047991 (Pubmed \| NEWS) Manual	Phase 2	Advanced Pancreatic Adenocarcinoma	117	NALIRIFOX (liposomal irinotecan+ 5-FU+leucovorin+oxaliplatin)	nelswpminw(eknrrqyoen) = oqmizhohbe xoijkfhxqc (afehaeikym, 5.52 - 9.23) View more	Positive	16 Jan 2026
				gemcitabine + nab-paclitaxel	nelswpminw(eknrrqyoen) = goiotvwlde xoijkfhxqc (afehaeikym, 3.38 - 5.32) View more
NCT04539808 (NeoOPTIMIZE, CTgov)	Phase 2	Pancreatic adenocarcinoma \| Pancreatic Ductal Adenocarcinoma	42	mFOLFIRINOX+nab-paclitaxel+leucovorin calcium+gemcitabine hydrochloride+potassium+losartan+Oxaliplatin+irinotecan hydrochloride+capecitabine+fluorouracil	lknyefhlzb = lsqucvzjpx sdkanxfzcv (bjaifljiit, zfmaoibffk - ivarcuylys) View more	-	14 Jan 2026
JPRN-jRCTs031200094 (ASCO_GI2026)	Phase 2	Esophageal Squamous Cell Carcinoma Neoadjuvant	53	Neoadjuvant fluorouracil, leucovorin, oxaliplatin, and docetaxel (FLOT) chemotherapy	izkbfzuckv(vsqqerykfn) = ojaexeiiwr kigjxwguzm (osxiqsczim ) View more	Positive	08 Jan 2026
				Neoadjuvant fluorouracil, leucovorin, oxaliplatin, and docetaxel (FLOT) chemotherapy (without SCLN metastasis)	izkbfzuckv(vsqqerykfn) = dwqwgszvtm kigjxwguzm (osxiqsczim ) View more

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