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Gemcitabine monotherapy

AstraZeneca · Phase 3 active Small molecule ✓ Verified May 2026

Gemcitabine monotherapy is a Nucleoside analog; antimetabolite Small molecule drug developed by AstraZeneca. It is currently in Phase 3 development for Metastatic pancreatic cancer, Non-small cell lung cancer, Bladder cancer. Also known as: Background Gemcitabine-based Chemotherapy Regimen, GEMXAR.

Gemcitabine is a nucleoside analog that inhibits ribonucleotide reductase and gets incorporated into DNA, causing chain termination and cell death.

Gemcitabine is a small molecule used as a monotherapy for various conditions, including advanced solid tumors, nasopharyngeal carcinoma, non-muscle invasive bladder cancer, and carcinoma in situ of the bladder. It is administered as a treatment for these conditions, although the specific indications and dosing regimens may vary depending on the clinical trial or study.

Likelihood of approval
64.3% vs 58.3% industry baseline
If approved by FDA: likely 2028–2030
Steps remaining: NDA/BLA submission
Confidence: High
Why this estimate
  • 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.
  • Big-pharma sponsor +3.0pp
    AstraZeneca is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
Predicted approval windows by jurisdiction (conditional on FDA approval)
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 nameGemcitabine monotherapy
Also known asBackground Gemcitabine-based Chemotherapy Regimen, GEMXAR
SponsorAstraZeneca
Drug classNucleoside analog; antimetabolite
TargetRibonucleotide reductase; DNA incorporation
ModalitySmall molecule
Therapeutic areaOncology
PhasePhase 3

Mechanism of action

Gemcitabine is a deoxycytidine analog that is phosphorylated intracellularly to its active triphosphate form. It inhibits ribonucleotide reductase, reducing deoxyribonucleotide pools, and is incorporated into DNA where it causes chain termination and apoptosis. It is particularly effective against rapidly dividing cancer cells.

Approved indications

Common side effects

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

SourceUsed for
ClinicalTrials.govTrial 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:

Frequently asked questions about Gemcitabine monotherapy

What is Gemcitabine monotherapy?

Gemcitabine monotherapy is a Nucleoside analog; antimetabolite drug developed by AstraZeneca, indicated for Metastatic pancreatic cancer, Non-small cell lung cancer, Bladder cancer.

How does Gemcitabine monotherapy work?

Gemcitabine is a nucleoside analog that inhibits ribonucleotide reductase and gets incorporated into DNA, causing chain termination and cell death.

What is Gemcitabine monotherapy used for?

Gemcitabine monotherapy is indicated for Metastatic pancreatic cancer, Non-small cell lung cancer, Bladder cancer, Breast cancer, Ovarian cancer.

Who makes Gemcitabine monotherapy?

Gemcitabine monotherapy is developed by AstraZeneca (see full AstraZeneca pipeline at /company/astrazeneca).

Is Gemcitabine monotherapy also known as anything else?

Gemcitabine monotherapy is also known as Background Gemcitabine-based Chemotherapy Regimen, GEMXAR.

What drug class is Gemcitabine monotherapy in?

Gemcitabine monotherapy belongs to the Nucleoside analog; antimetabolite class. See all Nucleoside analog; antimetabolite drugs at /class/nucleoside-analog-antimetabolite.

What development phase is Gemcitabine monotherapy in?

Gemcitabine monotherapy is in Phase 3.

What are the side effects of Gemcitabine monotherapy?

Common side effects of Gemcitabine monotherapy include Myelosuppression (neutropenia, thrombocytopenia, anemia), Nausea and vomiting, Fatigue, Fever, Rash, Dyspnea.

What does Gemcitabine monotherapy target?

Gemcitabine monotherapy targets Ribonucleotide reductase; DNA incorporation and is a Nucleoside analog; antimetabolite.

Related

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing