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AZD0120
AZD0120 is a FGFR1 inhibitor Biologic drug developed by AstraZeneca. It is currently in Phase 3 development for FGFR1-amplified or FGFR1-altered solid tumors. Also known as: GC012F.
AZD0120 is a selective inhibitor of fibroblast growth factor receptor 1 (FGFR1) that blocks aberrant signaling in cancer cells dependent on FGFR1 activation.
AZD0120 is a medication being studied in clinical trials for multiple conditions, including multiple myeloma, lupus erythematosus, and relapsed/refractory multiple myeloma. It is being evaluated in combination with other treatments, such as cyclophosphamide and fludarabine, in a phase I clinical trial.
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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. -
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.
| 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 | AZD0120 |
|---|---|
| Also known as | GC012F |
| Sponsor | AstraZeneca |
| Drug class | FGFR1 inhibitor |
| Target | FGFR1 (Fibroblast Growth Factor Receptor 1) |
| Modality | Biologic |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
FGFR1 is frequently dysregulated in certain cancers through gene amplification or translocation, driving proliferation and survival. AZD0120 selectively targets FGFR1 to inhibit downstream signaling pathways, inducing cell cycle arrest and apoptosis in FGFR1-dependent tumors. This approach is designed to provide clinical benefit in cancers with FGFR1 alterations while minimizing off-target effects.
Approved indications
- FGFR1-amplified or FGFR1-altered solid tumors
Common side effects
- Hyperphosphatemia
- Diarrhea
- Nausea
- Fatigue
- Retinal toxicity
Key clinical trials
- Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis (PHASE1)
- A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. (PHASE1, PHASE2)
- A Study Comparing AZD0120, a Dual-targeted CAR-T Against B-cell Maturation Antigen (BCMA) and CD19, Versus Standard Regimens in Participants With Relapsed Refractory Multiple Myeloma (DURGA-4) (PHASE3)
- A Phase I, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Immunogenicity, Pharmacodynamics, and Preliminary Efficacy of AZD0120 in Participants With Multiple Myeloma (DURGA-2) (PHASE1)
- A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE (PHASE1, PHASE2)
- A Study of AZD0120 in Autoimmune Diseases (PHASE1)
- An Open-label Study of AZD0120 in Adults With Multiple Sclerosis (PHASE1)
- CAR-T Therapy Targeting CD19 and BCMA in Highly Sensitized Kidney Transplant Participants (EARLY_PHASE1)
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:
- AZD0120 CI brief — competitive landscape report
- AZD0120 updates RSS · CI watch RSS
- AstraZeneca portfolio CI
Frequently asked questions about AZD0120
What is AZD0120?
How does AZD0120 work?
What is AZD0120 used for?
Who makes AZD0120?
Is AZD0120 also known as anything else?
What drug class is AZD0120 in?
What development phase is AZD0120 in?
What are the side effects of AZD0120?
What does AZD0120 target?
Related
- Drug class: All FGFR1 inhibitor drugs
- Target: All drugs targeting FGFR1 (Fibroblast Growth Factor Receptor 1)
- Manufacturer: AstraZeneca — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for FGFR1-amplified or FGFR1-altered solid tumors
- Also known as: GC012F
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing