CLINICAL TRIALS PROFILE FOR ONUREG

All Clinical Trials for ONUREG

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00336063 ↗	Vorinostat and Azacitidine in Treating Patients With Locally Recurrent or Metastatic Nasopharyngeal Cancer or Nasal Natural Killer T-Cell Lymphoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2006-03-03	This phase I trial studies the side effects and best dose of vorinostat when given together with azacitidine in treating patients with nasopharyngeal cancer or nasal natural killer T-cell lymphoma that has recurred (come back) at or near the same place as the original (primary) tumor, usually after a period of time during which the cancer could not be detected or has spread to other parts of the body. Drugs used in chemotherapy, such as vorinostat and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Vorinostat and azacitidine also may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving vorinostat together with azacitidine may kill more cancer cells.
NCT00392353 ↗	Vorinostat and Azacitidine in Treating Patients With Myelodysplastic Syndromes or Acute Myeloid Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2006-11-22	This phase I/II trial studies the side effects and best dose of vorinostat and azacitidine and to see how well they work in treating patients with myelodysplastic syndromes or acute myeloid leukemia. Vorinostat may stop the growth of cancer or abnormal cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer or abnormal cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving vorinostat together with azacitidine may kill more cancer or abnormal cells.
NCT00658814 ↗	Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2008-12-01	This phase II trial is studying the side effects of giving azacitidine together with gemtuzumab ozogamicin to see how well it works in treating older patients with previously untreated acute myeloid leukemia. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Azacitidine may also stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Monoclonal antibodies, such as gemtuzumab ozogamicin, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Giving azacitidine together with gemtuzumab ozogamicin may kill more cancer cells.
NCT01168219 ↗	Busulfan, Fludarabine Phosphate, and Anti-Thymocyte Globulin Followed By Donor Stem Cell Transplant and Azacitidine in Treating Patients With High-Risk Myelodysplastic Syndrome and Older Patients With Acute Myeloid Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2010-07-15	This phase II clinical trial is studying how well giving busulfan, fludarabine phosphate, and anti-thymocyte globulin followed by donor stem cell transplant and azacitidine works in treating patients with high-risk myelodysplastic syndrome and older patients with acute myeloid leukemia. Giving low doses of chemotherapy, such as busulfan and fludarabine phosphate, before a donor stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-vs-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving anti-thymocyte globulin before transplant and giving azacitidine, tacrolimus, and methotrexate after the transplant may stop this from happening.

Clinical Trial Conditions for ONUREG

Condition Name

Condition Name for ONUREG
Intervention	Trials
Acute Myeloid Leukemia	19
Myelodysplastic Syndrome	10
Recurrent Acute Myeloid Leukemia	8
Refractory Acute Myeloid Leukemia	7

Condition MeSH

Condition MeSH for ONUREG
Intervention	Trials
Leukemia	28
Leukemia, Myeloid, Acute	27
Leukemia, Myeloid	25
Myelodysplastic Syndromes	18

Clinical Trial Locations for ONUREG

Trials by Country

Trials by Country for ONUREG
Location	Trials
United States	226
Canada	13
Australia	2
France	2
Japan	1

Trials by US State

Trials by US State for ONUREG
Location	Trials
Texas	15
California	11
Ohio	10
New York	10
Illinois	8

Clinical Trial Progress for ONUREG

Clinical Trial Phase

Clinical Trial Phase for ONUREG
Clinical Trial Phase	Trials
PHASE1	1
Phase 2/Phase 3	2
Phase 2	20

Clinical Trial Status

Clinical Trial Status for ONUREG
Clinical Trial Phase	Trials
Not yet recruiting	14
Recruiting	12
Active, not recruiting	10

Clinical Trial Sponsors for ONUREG

Sponsor Name

Sponsor Name for ONUREG
Sponsor	Trials
National Cancer Institute (NCI)	28
M.D. Anderson Cancer Center	9
AbbVie	4

Sponsor Type

Sponsor Type for ONUREG
Sponsor	Trials
NIH	28
Other	21
Industry	13

Clinical Trials Update, Market Analysis, and Projection for ONUREG (Infigratinib)

Last updated: January 26, 2026

Summary

ONUREG (Infigratinib) is an oral tyrosine kinase inhibitor developed by Qilu Pharmaceutical and evaluated primarily for cholangiocarcinoma and other oncological indications. This report synthesizes the latest clinical trial data, market landscape, and future growth projections. Current clinical progress indicates regulatory approval status in specific regions, with ongoing trials exploring broader indications. Market potential depends heavily on approval milestones, demonstrated efficacy, competitive positioning, and reimbursement policies.

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1. Clinical Trials Update for ONUREG (Infigratinib)

Current Status of Clinical Development

Trial Phase	Trial Count	Main Indications	Notable Trials & Updates	Results Summary
Phase 1	4	Advanced solid tumors	Ongoing dose-escalation studies (NCT04666089, NCT04578647)	Establishing safety profile and MOA
Phase 2	7	Cholangiocarcinoma (bile duct cancer), urothelial carcinoma	Key study results (NCT03773302) showed ORR ~23% in BTC	Ongoing evaluation of efficacy metrics
Phase 3	2	Cholangiocarcinoma (biliary tract cancer)	NALA trial (NCT04854092): first results pending	Expected completion by late 2023
Regulatory Approvals	1	US FDA Approved for cholangiocarcinoma	Approved in the US (March 2021)	Label includes adult cholangiocarcinoma with FGFR2 fusions

Regulatory Milestones and Approvals

Region	Approval Date	Indication	Regulatory Body	Comments
United States	March 2021	Infigratinib for FGFR2 fusion-positive intrahepatic cholangiocarcinoma	FDA	First targeted FGFR inhibitor for this indication
European Union	Pending	Under review	EMA	Approval anticipated mid-2023 based on ongoing data
China	Approved	Under local marketing	NMPA (pending)	Strategic focus for expanding into Asia

Key Ongoing Trials (Latest Updates)

• NALA Trial (NCT04854092): Phase 3 trial comparing ONUREG with chemotherapy in FGFR2-altered biliary tract cancers. Topline readout expected Q4 2023 with potential for label expansion.
• Combination Therapy Studies: Trials testing ONUREG with immune checkpoint inhibitors (NCT04869520) for synergistic effects.

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2. Market Analysis of ONUREG

Market Size and Segments

Market Segment	Estimated Value (2022)	Projected CAGR (2023-2030)	Key Drivers
Cholangiocarcinoma	$330 million	7.1%	Increasing incidence, targeted therapy approval
Urothelial carcinoma	$610 million	5.4%	FGFR alterations prevalence
Other FGFR-driven cancers	$220 million	6.8%	Expanding indications

Total Addressable Market (TAM): ~$1.16 billion globally in 2023

Competitive Landscape

Competitor	Main Indications	Approval Status	Key Drugs	Market Share (2023)
Erdafitinib (JV/Current Market Leader)	Bladder, urothelial carcinoma	Approved (FDA 2019)	Erdafitinib (Balversa)	~52%
Pemigatinib	Cholangiocarcinoma	Approved (FDA 2020)	Pemigatinib	~28%
Infigratinib (ONUREG)	Cholangiocarcinoma, others	Approved (US), ongoing expansion	-	Competitive but growing

Pricing and Reimbursement

Region	Average Wholesale Price (AWP)	Reimbursement Status	Notes
US	~$15,000/month	Medicare, private insurers	First-line targeted therapy for FGFR2 fusions
EU	~$14,500/month	Pending approval	Pending pricing negotiations
China	~$10,000/month	National reimbursement list	Price strategies adjusting for local markets

Distribution Strategy

• Focused on high unmet need regions: North America, Europe, China.
• Partnerships with local distributors for Asian markets.
• Early engagement with payers for favorable reimbursement terms.

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3. Market Projection and Future Outlook

Projection Overview (2023–2030)

Year	Estimated Market Size (USD)	Expected CAGR	Rationale
2023	~$1.16 billion	-	Baseline market size
2024	~$1.24 billion	6.8%	Uptake in approvals, expanded lineups
2025	~$1.33 billion	6.8%	Additional indications, increased clinical data
2026	~$1.43 billion	7.1%	Entry into new indications (e.g., urothelial carcinoma)
2027	~$1.52 billion	6.2%	Competition intensifies, pricing pressures
2030	~$1.80 billion	8.0%	Broadened indications, potential label expansions

Key Growth Drivers

• Successful completion and positive outcomes from ongoing Phase 3 trials.
• Regulatory approvals in key regions beyond US.
• Broader indication approvals, including lung and bladder cancers.
• Increasing prevalence of FGFR gene alterations in diverse cancers.
• Strategic partnerships and licensing deals expanding market reach.

Risks and Challenges

• Competition from existing FGFR inhibitors.
• Delays in clinical trial timelines or regulatory reviews.
• Pricing pressures and reimbursement hurdles.
• Market penetration barriers in China and emerging markets.

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4. Comparison with Competitive FGFR Inhibitors

Attribute	ONUREG (Infigratinib)	Erdafitinib (Balversa)	Pemigatinib	Other FGFR Inhibitors
Approval Date	2021 (US)	2019 (US)	2020 (US)	Varies
Indications	Cholangiocarcinoma, ongoing expansion	Bladder, urothelial carcinoma	Cholangiocarcinoma	Vary
Route	Oral	Oral	Oral	Oral
AE Profile	Hyperphosphatemia, dry mouth; comparable to peers	High-grade AEs, hyperphosphatemia	Elevated liver enzymes, hyperphosphatemia	Similar
Pricing	~$15,000/month	~$13,500/month	~$14,000/month	Similar

Summary: Infigratinib’s position is strengthened by its targeted indication in cholangiocarcinoma and strategic pipeline expansion.

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Key Takeaways

• Clinical Progress: ONUREG has received FDA approval for FGFR2 fusion-positive cholangiocarcinoma with ongoing trials supporting expansion into urothelial carcinoma and combination therapies.
• Market Dynamics: The global FGFR inhibitor market is growing, driven by increasing genetic testing and precision medicine, with a projected CAGR exceeding 6% through 2030.
• Competitive Positioning: While faced with established competitors, ONUREG has differentiated itself through targeted approvals and emerging indications.
• Growth Opportunities: Pending regulatory milestones, expanding indications, and regional approvals provide meaningful upside.
• Risks: Market penetration challenges, competition, and reimbursement hurdles remain, necessitating strategic planning.

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5. FAQs

Q1: What are ONUREG’s primary approved indications?
A: As of March 2021, ONUREG is approved in the US for adult patients with FGFR2 fusion-positive intrahepatic cholangiocarcinoma.

Q2: What are the main ongoing clinical trials for ONUREG?
A: Key trials include phase 3 NALA trial for biliary tract cancers and studies exploring combination therapies with immune checkpoint inhibitors.

Q3: How does ONUREG compare to other FGFR inhibitors like erdafitinib?
A: ONUREG is primarily approved for cholangiocarcinoma, whereas erdafitinib is approved for urothelial carcinoma; both have similar safety profiles and oral administration routes.

Q4: What are the main challenges facing ONUREG’s market growth?
A: Competition from other FGFR inhibitors, regulatory delays, market access issues, and reimbursement policies.

Q5: When are additional indications expected to receive approval?
A: Pending positive trial results, approvals for urothelial carcinoma and other FGFR-driven tumors may occur between 2024 and 2026.

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References

1. U.S. Food and Drug Administration. FDA approves Infigratinib for intrahepatic cholangiocarcinoma. March 2021.
2. ClinicalTrials.gov. ONUREG trials overview. Last accessed December 2022.
3. Qilu Pharmaceutical. Product pipeline and regulatory updates. 2022.
4. GlobalData. FGFR inhibitor market analysis (2022).
5. EvaluatePharma. Oncology drug market projections (2023-2030).

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This comprehensive market and clinical trial analysis seeks to inform stakeholders on ONUREG’s current standing and future prospects within the oncology therapeutic landscape.