CLINICAL TRIALS PROFILE FOR ERDAFITINIB

All Clinical Trials for erdafitinib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02365597 ↗	An Efficacy and Safety Study of Erdafitinib (JNJ-42756493) in Participants With Urothelial Cancer	Recruiting	Janssen Research & Development, LLC	Phase 2	2015-04-22	The purpose of this study is to evaluate the objective response rate (complete response [CR]+ partial response [PR]) of the selected dose regimen in participants with metastatic or surgically unresectable urothelial cancers that harbor specific FGFR genomic alterations.
NCT02421185 ↗	Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of JNJ-42756493 (Erdafitinib) in Participants With Advanced Hepatocellular Carcinoma	Completed	Janssen Research & Development, LLC	Phase 1/Phase 2	2015-05-25	The purpose of this study is to determine recommended Phase 2 dose [RP2D]) and the objective response rate of JNJ-42756493 (erdafitinib) in advanced hepatocellular carcinoma (HCC) participants with fibroblast growth factor (FGF) 19 amplification.
NCT02465060 ↗	Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)	Recruiting	National Cancer Institute (NCI)	Phase 2	2015-08-12	This phase II MATCH trial studies how well treatment that is directed by genetic testing works in patients with solid tumors or lymphomas that have progressed following at least one line of standard treatment or for which no agreed upon treatment approach exists. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic abnormalities (such as mutations, amplifications, or translocations) may benefit more from treatment which targets their tumor's particular genetic abnormality. Identifying these genetic abnormalities first may help doctors plan better treatment for patients with solid tumors, lymphomas, or multiple myeloma.
NCT02699606 ↗	A Study to Evaluate the Clinical Efficacy of JNJ-42756493 (Erdafitinib), A Pan-Fibroblast Growth Factor Receptor (FGFR) Tyrosine Kinase Inhibitor, In Asian Participants With Advanced Non-Small-Cell Lung Cancer, Urothelial Cancer, Esophageal Cancer O	Active, not recruiting	Janssen Research & Development, LLC	Phase 2	2016-07-08	The primary purpose of this study is to evaluate objective response rate (ORR) as per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 of erdafitinib in a molecularly-defined subset of Asian participants with non-small-cell lung cancer (NSCLC), urothelial cancer, esophageal cancer and cholangiocarcinoma.
NCT02925234 ↗	The Drug Rediscovery Protocol (DRUP Trial)	Recruiting	Amgen	Phase 2	2016-08-01	This is a prospective, non-randomized clinical trial that aims to describe the efficacy and toxicity of commercially available, targeted anticancer drugs* prescribed for treatment of patients with advanced cancer with a potentially actionable variant as revealed by a genomic or protein expression test. The study also aims to simplify patient access to approved targeted therapies that are contributed to the program by collaborating pharmaceutical companies and to perform next generation sequencing on tumor biopsies for biomarker analyses. Eligible patients have an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma for which standard treatment options are no longer available and acceptable performance status and organ function. A genomic or protein expression test must have been performed on the tumor and the results must identify at least one potentially actionable molecular variant as defined in the protocol. Results from the molecular profiling test will be used to determine an appropriate drug(s) from among those available in the protocol. The choice of drug will be supported by a list of potential profiles, a molecular tumor board, a knowledge library and by study coordinators for review and approval of the match. The protocol-specified treatment will be administered to the patient once any drug-specific eligibility criteria are confirmed and a fresh pre-treatment biopsy is performed for future genetic studies. All patients who receive treatment with a drug available in the protocol will be followed for standard efficacy outcomes including tumor response, progression-free and overall survival as well as duration of treatment. In addition, treatment related toxicity will be evaluated.

Clinical Trial Conditions for erdafitinib

Condition Name

Condition Name for erdafitinib
Intervention	Trials
Advanced Malignant Solid Neoplasm	4
Refractory Malignant Solid Neoplasm	4
Neoplasm	3
Recurrent Malignant Solid Neoplasm	3

Condition MeSH

Condition MeSH for erdafitinib
Intervention	Trials
Urinary Bladder Neoplasms	7
Neoplasms	6
Carcinoma	6
Carcinoma, Transitional Cell	4

Clinical Trial Locations for erdafitinib

Trials by Country

Trials by Country for erdafitinib
Location	Trials
United States	285
Germany	12
Spain	10
France	8
Belgium	8

Trials by US State

Trials by US State for erdafitinib
Location	Trials
Texas	12
Pennsylvania	12
New York	11
North Carolina	10
Illinois	10

Clinical Trial Progress for erdafitinib

Clinical Trial Phase

Clinical Trial Phase for erdafitinib
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	3
Phase 3	1

Clinical Trial Status

Clinical Trial Status for erdafitinib
Clinical Trial Phase	Trials
Recruiting	16
Not yet recruiting	6
COMPLETED	4

Clinical Trial Sponsors for erdafitinib

Sponsor Name

Sponsor Name for erdafitinib
Sponsor	Trials
Janssen Research & Development, LLC	14
National Cancer Institute (NCI)	10
University Health Network, Toronto	2

Sponsor Type

Sponsor Type for erdafitinib
Sponsor	Trials
Industry	35
Other	11
NIH	10

Clinical Trials Update, Market Analysis, and Projection for Erdafitinib

Last updated: October 29, 2025

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Introduction

Erdafitinib (marketed as Balversa by Janssen) is a targeted therapeutic agent approved for the treatment of locally advanced or metastatic urothelial carcinoma harboring specific fibroblast growth factor receptor (FGFR) alterations. As a potent FGFR kinase inhibitor, erdafitinib represents a significant advancement in precision oncology. Its development and commercialization landscape are evolving amidst ongoing clinical research, competition, and expanding indications. This report synthesizes recent clinical trial data, market dynamics, and future projections for erdafitinib.

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Clinical Trials Landscape and Developments

Regulatory Approval and Key Trials

Erdafitinib received accelerated approval from the FDA in April 2019 for advanced urothelial carcinoma with FGFR alterations, based primarily on the phase 2 BLC2001 trial. This pivotal study demonstrated an objective response rate (ORR) near 40% among FGFR-mutated patients, with a manageable safety profile [1].

Since approval, the drug has undergone several clinical trials to expand its indications and optimize dosing and safety profiles:

• FIREFLY-1 (NCT03624195): A phase 2 trial evaluating erdafitinib’s efficacy in combination with other agents, including cetrelimab (a PD-1 inhibitor), for advanced urothelial carcinoma. Preliminary results indicated promising synergistic effects, prompting further investigation.

• FGFR-Targeted Therapy Trials: Multiple ongoing phase 3 trials are assessing erdafitinib’s efficacy compared to standard chemotherapies or immunotherapies in FGFR-altered urothelial carcinoma. Results are expected to clarify its position in frontline settings.

• Trials in Other Cancers: Although primarily approved for bladder cancer, erdafitinib is under investigation for other malignancies with FGFR alterations, such as cholangiocarcinoma and gastric cancers. Notably, a phase 2 trial (NCT03834220) is exploring its efficacy in cholangiocarcinoma with FGFR2 fusions.

Emerging Data and Safety Profile

Recent updates from clinical trials indicate that erdafitinib maintains a consistent safety profile, with common adverse events including hyperphosphatemia, dry mouth, diarrhea, and dry skin. Grade 3 or higher adverse events remain manageable with dose modifications [2].

The ongoing studies aim to refine patient selection, combination strategies, and dose optimization to improve response rates and minimize toxicity.

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Market Analysis

Current Market Landscape

The global market for FGFR inhibitors is witnessing rapid growth driven by the expanding understanding of FGFR pathway aberrations across multiple cancers:

• Key Players: Besides erdafitinib, other FGFR inhibitors include infigratinib (Incyte), pemigatinib (Incyte), and futibatinib (Taiho). The competitive landscape involves both targeted therapies and companion diagnostic developments.

• Market Size: The urothelial carcinoma drugs market was valued at approximately USD 2.3 billion in 2022, projected to grow at a CAGR of around 8% through 2030 [3]. FGFR-targeted agents constitute a significant share, driven by the subset of patients with FGFR mutations (~15-20%).

• Pricing and Reimbursement: Erdafitinib’s cost remains high, with list prices exceeding USD 15,000 per month. Reimbursement strategies, especially in Europe and Asia, hinge on local health technology assessments and biomarker testing coverage.

Key Market Drivers

• Precision Medicine: Increased utilization of FGFR testing enhances patient stratification, widening erdafitinib’s eligible population.
• Regulatory Expansions: New approvals, especially in combination regimens or additional indications, broaden market access.
• Biomarker Testing Advancements: Development of robust diagnostic tools (e.g., FGFR-specific NGS panels) improve detection rates and treatment planning.

Market Challenges

• Resistance Mechanisms: Acquired resistance to FGFR inhibition (~20-30%) challenges continued efficacy and necessitates combination approaches or alternative inhibitors.
• Adverse Events Management: Toxicity management remains pivotal; severe side effects can limit patient adherence.
• Competitive Pressure: The advent of other FGFR inhibitors with distinct attributes (e.g., futibatinib’s irreversible binding) may impact erdafitinib's market share.

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Market Projection and Future Outlook

Growth Forecasts

Analysts project the FGFR inhibitor market to grow at a CAGR of over 10% in the next decade, driven initially by urothelial carcinoma and potentially extending into other malignancies:

• 2023-2030 Market Value: Estimated to reach USD 8-10 billion across all FGFR inhibitors; erdafitinib’s share is expected to constitute approximately USD 1.5-3 billion, factoring in current uptake, emerging data, and expanded indications.

• Geographical Expansion: Europe and Asia-Pacific present significant growth opportunities, contingent on regulatory approvals and diagnostic integration.

Strategic Opportunities

• Combination Therapies: Combining erdafitinib with immunotherapies (e.g., PD-1/PD-L1 inhibitors) could increase response rates and durability, opening new revenue streams.
• Biomarker Innovation: Enhanced FGFR mutation detection methods can expand eligible patient pools.
• New Indications: Accelerated trials in colorectal, cholangiocarcinoma, and other FGFR-driven tumors may diversify revenue sources.

Risks and Uncertainties

• Clinical Efficacy: Variability in response across tumor types could limit broad applicability.
• Regulatory Delays: Pending trial results might delay approvals for new indications.
• Pricing Pressures: Cost-containment measures and pricing negotiations may constrain profit margins.

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Conclusion

Erdafitinib continues to cement its role as a targeted therapy for FGFR-driven urothelial carcinoma. Its clinical profile and market position are poised for expansion pending positive trial outcomes, combination strategy development, and regulatory approvals for additional indications. Market dynamics indicate robust growth potential, albeit tempered by competition, resistance mechanisms, and safety management challenges.

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Key Takeaways

• Ongoing clinical trials aim to expand erdafitinib’s indications and optimize its combination with immunotherapies, potentially transforming its utility in oncology.
• The FGFR inhibitor market is expanding rapidly, with erdafitinib positioned as a leading agent due to its early FDA approval and strong clinical data.
• Market growth hinges on advancements in diagnostic biomarker testing, regulatory approvals in new regions, and strategic combination therapies.
• Resistance and toxicity management remain critical to maintaining efficacy and market competitiveness.
• Strategic investments in diagnostics, combination trial designs, and global expansion will be crucial for stakeholders aiming to capitalize on erdafitinib's market potential.

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FAQs

1. What recent clinical developments have been reported for erdafitinib?
Recent trials have shown promising activity in combination with immunotherapies and expanded studies in other FGFR-altered malignancies like cholangiocarcinoma. These efforts aim to broaden its therapeutic reach beyond urothelial carcinoma.

2. How does erdafitinib compare with other FGFR inhibitors?
Erdafitinib’s early FDA approval, safety profile, and specificity position it favorably. However, competitors like futibatinib, with irreversible binding, and pemigatinib may offer differences in efficacy, toxicity, or resistance profiles, influencing market dynamics.

3. What are key challenges facing erdafitinib’s market growth?
Resistance development, toxicity management, high pricing, and competitive pressures present ongoing hurdles that may affect adoption and revenue.

4. Which markets are expected to drive future growth for erdafitinib?
The U.S., Europe, and Asia-Pacific are pivotal, with expanding use driven by diagnostic improvements and regulatory approvals, especially as combination strategies mature.

5. What is the outlook for erdafitinib in non-urothelial cancers?
Early clinical evidence suggests potential in other FGFR-driven cancers, but validation requires ongoing trials. Success here could significantly diversify revenue streams.

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References

[1] Turner, N. et al., "Erdafitinib in locally advanced or metastatic urothelial carcinoma," The New England Journal of Medicine, 2019.
[2] Singh, S. et al., "Safety and efficacy profile of erdafitinib," Oncologist, 2021.
[3] MarketsandMarkets, "FGFR Inhibitors Market," 2022.

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Note: All data are projected figures and trends as of early 2023; ongoing trials and evolving market conditions may influence future outcomes.