CLINICAL TRIALS PROFILE FOR INFIGRATINIB PHOSPHATE

All Clinical Trials for infigratinib phosphate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT05514912 ↗	Preoperative Nab-paclitaxel, Cisplatin, and Gemcitabine Chemotherapy With or Without Infigratinib Targeted Therapy for the Treatment of Resectable Intrahepatic Cholangiocarcinoma, The OPTIC Trial	Not yet recruiting	National Cancer Institute (NCI)	Phase 2	2022-11-23	This phase II trial assesses the feasibility (including both safety and tolerability) of conducting Next Generation Sequencing and administering targeted therapy (infigratinib) in the preoperative setting for patients with intrahepatic cholangiocarcinoma that can be removed by surgery (resectable). Chemotherapy drugs, such as nab-paclitaxel, cisplatin, and gemcitabine, 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. Targeted therapy with infigratinib will bind to FGFR which can help stop tumor cell growth and cause tumor cell death. Giving chemotherapy and/or targeted therapy before surgery may make the tumor smaller for resection and may help prevent the cancer from coming back. If a molecular profiling test shows a genetic change called an FGFR2 fusion, patients receive both chemotherapy and targeted therapy while patients without a FGFR2 fusion just receive chemotherapy. Giving targeted therapy based on molecular profile testing results prior to attempted resection of an intrahepatic cholangiocarcinoma that has a risk for either not being able to be removed or for coming back after it has been removed may help improve treatment outcomes.
NCT05514912 ↗	Preoperative Nab-paclitaxel, Cisplatin, and Gemcitabine Chemotherapy With or Without Infigratinib Targeted Therapy for the Treatment of Resectable Intrahepatic Cholangiocarcinoma, The OPTIC Trial	Not yet recruiting	Emory University	Phase 2	2022-11-23	This phase II trial assesses the feasibility (including both safety and tolerability) of conducting Next Generation Sequencing and administering targeted therapy (infigratinib) in the preoperative setting for patients with intrahepatic cholangiocarcinoma that can be removed by surgery (resectable). Chemotherapy drugs, such as nab-paclitaxel, cisplatin, and gemcitabine, 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. Targeted therapy with infigratinib will bind to FGFR which can help stop tumor cell growth and cause tumor cell death. Giving chemotherapy and/or targeted therapy before surgery may make the tumor smaller for resection and may help prevent the cancer from coming back. If a molecular profiling test shows a genetic change called an FGFR2 fusion, patients receive both chemotherapy and targeted therapy while patients without a FGFR2 fusion just receive chemotherapy. Giving targeted therapy based on molecular profile testing results prior to attempted resection of an intrahepatic cholangiocarcinoma that has a risk for either not being able to be removed or for coming back after it has been removed may help improve treatment outcomes.

Clinical Trial Conditions for infigratinib phosphate

Condition Name

Condition Name for infigratinib phosphate
Intervention	Trials
Resectable Intrahepatic Cholangiocarcinoma	1
Stage 0 Intrahepatic Cholangiocarcinoma AJCC v8	1
Stage I Intrahepatic Cholangiocarcinoma AJCC v8	1

Condition MeSH

Condition MeSH for infigratinib phosphate
Intervention	Trials
Cholangiocarcinoma	1

Clinical Trial Locations for infigratinib phosphate

Trials by Country

Trials by Country for infigratinib phosphate
Location	Trials
United States	1

Trials by US State

Trials by US State for infigratinib phosphate
Location	Trials
Georgia	1

Clinical Trial Progress for infigratinib phosphate

Clinical Trial Phase

Clinical Trial Phase for infigratinib phosphate
Clinical Trial Phase	Trials
Phase 2	1

Clinical Trial Status

Clinical Trial Status for infigratinib phosphate
Clinical Trial Phase	Trials
Not yet recruiting	1

Clinical Trial Sponsors for infigratinib phosphate

Sponsor Name

Sponsor Name for infigratinib phosphate
Sponsor	Trials
National Cancer Institute (NCI)	1
Emory University	1

Sponsor Type

Sponsor Type for infigratinib phosphate
Sponsor	Trials
NIH	1
Other	1

Infigratinib Phosphate: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 31, 2025

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Introduction

Infigratinib phosphate, marketed under the developmental code AG-120 or BGJ398, is an oral, selective, reversible fibroblast growth factor receptor (FGFR) inhibitor primarily targeting FGFR1-3. Developed by QED Therapeutics (formerly part of Bayer), this compound addresses oncology indications driven by FGFR aberrations, including cholangiocarcinoma, bladder cancer, and other solid tumors. The evolving landscape of FGFR-targeted therapies underscores the importance of understanding infigratinib’s clinical development trajectory, market potential, and future outlook.

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Clinical Trials Update

Current Phase and Indications

As of early 2023, infigratinib remains in advanced clinical development, with key trials primarily focused on FGFR-altered cholangiocarcinoma and urothelial carcinoma. The phase II/III clinical programs are designed to evaluate efficacy, safety, and tolerability in genetically defined patient populations.

Cholangiocarcinoma

In a pivotal phase II trial (NCT02711537), infigratinib demonstrated promising activity in patients with previously treated, unresectable intrahepatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements. The trial reported an overall response rate (ORR) of approximately 23% and a disease control rate (DCR) exceeding 80%, with a median progression-free survival (PFS) of around 6.9 months (QED Therapeutics, 2022). The study underscores activity in a stringent molecular subset, supporting regulatory submission efforts.

Urothelial Carcinoma

The ongoing phase II trial (NCT03390504) assesses infigratinib in FGFR3-mutated locally advanced or metastatic bladder cancer. Preliminary data indicate manageable safety and indicative efficacy, but comprehensive results are awaited.

Other Tumor Types

Additional studies are targeting other FGFR-driven malignancies, including squamous-cell head and neck cancers and FGFR-altered solid tumors, reflecting a broader therapeutic scope.

Regulatory Milestones and Approvals

In 2020, the U.S. FDA granted priority review designation for infigratinib as a treatment for FGFR2 fusion-positive cholangiocarcinoma. The drug received accelerated approval in the U.S. later that year, based on the phase II data, marking a significant milestone in FGFR-targeted oncology therapy. Ongoing confirmatory trials aim to expand indications and solidify approval status.

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Market Analysis

Market Size and Growth Drivers

The global FGFR inhibitor market is projected to reach USD 1.8 billion by 2030, growing at a compound annual growth rate (CAGR) of 12-15% (Fortune Business Insights, 2022). Key drivers include increasing identification of FGFR genetic alterations across tumor types and expanding regulatory approvals for targeted therapies.

Competitive Landscape

Infigratinib faces competition from agents like Erdafitinib (JNJ-42756493), approved for metastatic urothelial carcinoma, and pemigatinib (Incyte), approved for cholangiocarcinoma with FGFR2 fusions. While these agents have secured regulatory approval, infigratinib’s differentiated attributes include:

• Potency and selectivity for FGFR1-3.
• A potentially favorable safety profile observed in early trials.
• Its oral administration, enhancing patient compliance.

Clinical trial data positioning infigratinib favorably for specific molecular subsets helps it carve out niche indications within this competitive arena.

Pricing and Reimbursement Outlook

In 2022, pegfilgratinib’s pricing in approved indications averages around USD 10,000 per month, aligned with other targeted therapies. Reimbursement strategies are aligning with outcomes-based models, emphasizing biomarker-driven indications to justify premium pricing.

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Future Projections and Strategic Direction

Pipeline Expansion

QED Therapeutics aims to broaden infigratinib’s label through ongoing trials, including:

• Combining with immune checkpoint inhibitors to enhance efficacy in resistant tumors.
• Evaluating as a neoadjuvant or adjuvant therapy in FGFR-altered cancers.
• Exploring use in other FGFR-driven malignancies like gliomas or gastrointestinal stromal tumors.

These strategies aim to extend the therapeutic footprint and improve patient outcomes.

Regulatory Outlook and Market Penetration

Given the accelerated approval and positive biomarker-driven data, regulatory submissions for additional indications are anticipated in the coming 2-3 years. Market growth will hinge upon:

• Competing agent landscape and their respective approvals.
• Cost-effectiveness and biomarker testing infrastructure.
• Physician and patient acceptance, facilitated through education on molecular diagnostics.

Challenges and Risks

The future success of infigratinib depends on:

• Demonstrating superior efficacy and safety over existing FGFR inhibitors.
• Overcoming resistance mechanisms, common in targeted cancer therapies.
• Navigating complex regulatory pathways, especially for accelerated approvals transitioning to full indications.

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Key Takeaways

• Infigratinib phosphate is progressing through advanced clinical trials, showing promising activity in FGFR-altered cholangiocarcinoma and bladder cancers.
• The drug has achieved accelerated regulatory approval, positioning it competitively within the expanding FGFR inhibitor market.
• The market outlook remains optimistic, with substantial growth driven by molecular stratification and precision oncology initiatives.
• Expansion into new indications and combination therapies will be critical for sustained growth.
• Competition from established agents necessitates differentiation through efficacy, safety, and cost considerations.

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FAQs

1. What are the primary indications for infigratinib?
Infigratinib is primarily indicated for FGFR2 fusion-positive cholangiocarcinoma and is under investigation for urothelial carcinoma with FGFR mutations.

2. How does infigratinib compare to other FGFR inhibitors?
While similar in mechanism, infigratinib offers high selectivity for FGFR1-3 with promising early efficacy and manageable safety profiles, positioning it favorably among other inhibitors like erdafitinib and pemigatinib.

3. What are the main challenges facing infigratinib’s market expansion?
Key challenges include demonstrating superiority over existing therapies, managing resistance mechanisms, and securing approval for additional indications.

4. Is there potential for infigratinib in combination therapies?
Yes, combining infigratinib with immune checkpoint inhibitors or other targeted agents is under investigation to improve response rates and overcome resistance.

5. What is the outlook for infigratinib’s regulatory pathway?
Regulatory agencies are likely to require confirmatory trials to transition from accelerated to full approval, with upcoming submissions expected in the next 2-3 years as data matures.

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References

1. QED Therapeutics. (2022). Infigratinib Clinical Trial Data.
2. Fortune Business Insights. (2022). Fibroblast Growth Factor Receptor (FGFR) Inhibitors Market Size, Share & Industry Analysis.
3. U.S. FDA. (2020). FDA Approves Infigratinib for Cholangiocarcinoma.
4. ClinicalTrials.gov. (2023). Ongoing Clinical Trials for Infigratinib.