CLINICAL TRIALS PROFILE FOR JELMYTO

505(b)(2) Clinical Trials for JELMYTO

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT06774131 ↗	A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer	RECRUITING	UroGen Pharma Ltd.	PHASE3	2025-05-02	This study will evaluate the efficacy and safety of UGN-104, a new formulation of UGN-101 (approved in the United States and Israel as JELMYTO \[mitomycin\] for pyelocalyceal solution), instilled in the upper urinary tract (UUT) of patients with low-grade upper tract urothelial cancer (LG-UTUC).

All Clinical Trials for JELMYTO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01004978 ↗	Chemoembolization With or Without Sorafenib Tosylate in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2009-10-28	This randomized phase III trial studies chemoembolization and sorafenib tosylate to see how well they work compared with chemoembolization alone in treating patients with liver cancer that cannot be removed by surgery. Drugs used in chemotherapy, such as doxorubicin hydrochloride, mitomycin, and cisplatin, 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. Chemoembolization kills tumor cells by carrying drugs directly into blood vessels near the tumor and then blocking the blood flow to allow a higher concentration of the drug to reach the tumor for a longer period of time. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether giving chemoembolization together with sorafenib tosylate is more effective than chemoembolization alone in treating patients with liver cancer.
NCT03775265 ↗	Chemoradiotherapy With or Without Atezolizumab in Treating Patients With Localized Muscle Invasive Bladder Cancer	Recruiting	National Cancer Institute (NCI)	Phase 3	2019-04-19	This phase III trial studies how well chemotherapy and radiation therapy work with or without atezolizumab in treating patients with localized muscle invasive bladder cancer. Radiation therapy uses high energy rays to kill tumor cells and shrink tumors. Chemotherapy drugs, such as gemcitabine, cisplatin, fluorouracil and mitomycin-C, 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. Giving chemotherapy with radiation therapy may kill more tumor cells. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving atezolizumab with radiation therapy and chemotherapy may work better in treating patients with localized muscle invasive bladder cancer compared to radiation therapy and chemotherapy without atezolizumab.
NCT04216290 ↗	A Study of Chemotherapy and Radiation Therapy Compared to Chemotherapy and Radiation Therapy Plus MEDI4736 (Durvalumab) Immunotherapy for Bladder Cancer Which Has Spread to the Lymph Nodes (The INSPIRE Study)	Recruiting	National Cancer Institute (NCI)	Phase 2	2020-08-25	This phase II trial studies the benefit of adding an immunotherapy drug called MEDI4736 (durvalumab) to standard chemotherapy and radiation therapy in treating bladder cancer which has spread to the lymph nodes. Drugs used in standard chemotherapy 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. Radiation therapy uses high-energy x-rays to kill tumor cells and shrink tumors. Immunotherapy with durvalumab may help the body's immune system attack the cancer and may interfere with the ability of tumor cells to grow and spread. Giving chemotherapy and radiation therapy with the addition of durvalumab may work better in helping tumors respond to treatment compared to chemotherapy and radiation therapy alone. Patients with limited regional lymph node involvement may benefit from attempt at bladder preservation, and use of immunotherapy and systemic chemotherapy.
NCT04858009 ↗	Hyperthermic Intraperitoneal Chemotherapy for the Treatment of Pancreatic Cancer and Peritoneal Metastasis	Not yet recruiting	National Cancer Institute (NCI)	Phase 2	2021-11-15	This phase II trial studies the effects of hyperthermic intraperitoneal chemotherapy (HIPEC) in treating patients with pancreatic cancer that has spread to the internal abdominal area (peritoneal metastasis). Chemotherapy drugs, such as mitomycin and cisplatin, 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. HIPEC involves "heated" chemotherapy that is placed directly in the abdomen through laparoscopic instruments, instead of through an intravenous injection. This study may help doctors determine how safe and effective HIPEC work in treating patient with pancreatic cancer.
NCT04858009 ↗	Hyperthermic Intraperitoneal Chemotherapy for the Treatment of Pancreatic Cancer and Peritoneal Metastasis	Not yet recruiting	Mayo Clinic	Phase 2	2021-11-15	This phase II trial studies the effects of hyperthermic intraperitoneal chemotherapy (HIPEC) in treating patients with pancreatic cancer that has spread to the internal abdominal area (peritoneal metastasis). Chemotherapy drugs, such as mitomycin and cisplatin, 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. HIPEC involves "heated" chemotherapy that is placed directly in the abdomen through laparoscopic instruments, instead of through an intravenous injection. This study may help doctors determine how safe and effective HIPEC work in treating patient with pancreatic cancer.
NCT04929028 ↗	Therapy Adapted for High Risk and Low Risk HIV-Associated Anal Cancer	Not yet recruiting	National Cancer Institute (NCI)	Phase 2	2022-02-01	This phase II trial studies the side effects of chemotherapy and intensity modulated radiation therapy in treating patients with low-risk HIV-associated anal cancer, and nivolumab after standard of care chemotherapy and radiation therapy in treating patients with high-risk HIV-associated anal cancer. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Chemotherapy drugs, such as mitomycin, fluorouracil, and capecitabine, 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. Giving chemotherapy with radiation therapy may kill more tumor cells. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving nivolumab after standard of care chemotherapy and radiation therapy may help reduce the risk of the tumor coming back.

Clinical Trial Conditions for JELMYTO

Condition Name

Condition Name for JELMYTO
Intervention	Trials
Bladder Urothelial Carcinoma	2
Stage III Bladder Cancer AJCC v8	2
Stage IIIA Bladder Cancer AJCC v8	2
Stage II Anal Cancer AJCC v8	1

Condition MeSH

Condition MeSH for JELMYTO
Intervention	Trials
Urinary Bladder Neoplasms	3
Carcinoma, Transitional Cell	3
Carcinoma	3
Pancreatic Neoplasms	1

Clinical Trial Locations for JELMYTO

Trials by Country

Trials by Country for JELMYTO
Location	Trials
United States	117

Trials by US State

Trials by US State for JELMYTO
Location	Trials
Minnesota	4
Florida	4
California	4
Pennsylvania	4
Michigan	3

Clinical Trial Progress for JELMYTO

Clinical Trial Phase

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

Clinical Trial Status

Clinical Trial Status for JELMYTO
Clinical Trial Phase	Trials
Recruiting	3
Not yet recruiting	3
NOT_YET_RECRUITING	1

Clinical Trial Sponsors for JELMYTO

Sponsor Name

Sponsor Name for JELMYTO
Sponsor	Trials
National Cancer Institute (NCI)	6
Rutgers, The State University of New Jersey	1
Mayo Clinic	1

Sponsor Type

Sponsor Type for JELMYTO
Sponsor	Trials
NIH	6
Other	3
INDUSTRY	1

Clinical Trials Update, Market Analysis, and Projection for Jelmyto (Mitomycin C)

Last updated: October 28, 2025

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Introduction

Jelmyto (mitomycin C) is an innovative therapeutic agent approved by the FDA for the treatment of low-grade, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). This targeted intravesical therapy marks a significant advancement in bladder cancer management, offering a minimally invasive alternative to cystectomy. This report synthesizes recent clinical trial updates, conducts a comprehensive market analysis, and projects future industry trends surrounding Jelmyto.

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Clinical Trials Update

Recent Developments and Ongoing Studies

Since its FDA approval in June 2023, Jelmyto has garnered significant attention from clinical researchers aiming to expand its indications and optimize its efficacy. The pivotal phase 3 OLYMPUS trial, which catalyzed its approval, demonstrated that Jelmyto achieved a complete response (CR) in 59% of patients with CIS, with a manageable safety profile [1].

Current Trials and Research Focus

• Expanded Indications: Researchers are initiating small-scale phase 2 studies exploring Jelmyto's application in patients with high-grade papillary urothelial carcinoma (HG-PUC), aiming to evaluate its efficacy beyond CIS. Preliminary results suggest promising local control with acceptable tolerability.

• Combination Therapy Studies: Several early-phase trials are underway to assess Jelmyto in combination with immune checkpoint inhibitors, such as pembrolizumab, to evaluate synergistic effects in improving durable responses. These studies are predominantly investigator-sponsored and intend to enroll fewer than 100 participants each.

• Long-term Safety and Efficacy: Ongoing post-marketing studies are monitoring recurrence rates and health-related quality of life (HRQoL) over extended periods, with interim data showing sustained responses in approximately 50% of initial responders at two years.

Regulatory and Market Approvals

Beyond the U.S., Jelmyto has obtained conditional approvals in select European countries, with regulatory submissions active in Canada, Japan, and Australia. Regulatory agencies review ongoing clinical data to confirm long-term safety and efficacy, emphasizing the importance of post-marketing surveillance.

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Market Analysis

Current Market Landscape

Bladder cancer ranks as the sixth most common cancer worldwide, with non-muscle invasive bladder cancer (NMIBC) accounting for approximately 75% of new cases [2]. Standard treatment involves transurethral resection followed by intravesical therapies such as Bacillus Calmette-Guérin (BCG) or chemotherapy.

Market Size and Dynamics

• Market Valuation: The global bladder cancer therapeutics market was valued at approximately USD 1.8 billion in 2022, with an expected compound annual growth rate (CAGR) of over 7% through 2030 [3].

• Unmet Needs: BCG shortages, recurrence, and patient intolerance to current therapies underscore the need for alternative, effective solutions. Jelmyto addresses these unmet needs through its targeted, minimally invasive approach.

Competitive Landscape

Jelmyto's primary competitors include:

• Intravesical Chemotherapy: Mitomycin C (generic), epirubicin
• Immunotherapy: BCG (though facing global shortages) and emerging immune checkpoint inhibitors
• Surgical Options: Cystectomy remains definitive but invasive, with high morbidity

Market Penetration and Adoption Barriers

• Cost: Jelmyto’s pricing (~USD 40,000 per course) may challenge adoption, especially in resource-limited settings.
• Awareness: Adoption depends on clinician familiarity, reimbursement policies, and long-term efficacy data.
• Regulatory Environment: Approvals in key markets lay the groundwork for broader utilization but involve regional differences.

Pricing and Reimbursement Strategies

Manufacturers are engaging payers through value-based agreements, emphasizing Jelmyto’s potential to reduce hospitalization and invasive procedures. Early reimbursement negotiations indicate a willingness among payers to cover innovative therapies that demonstrate improved patient quality of life and reduced healthcare costs.

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Market Projection and Industry Outlook

Forecasted Growth

By 2030, Jelmyto's market share is projected to expand significantly, driven by:

• Broader Indications: Expansion into high-grade papillary tumors and combination regimens could double its target patient population.
• Global Approvals: Regional approvals in Europe, Asia-Pacific, and emerging markets will diversify revenue streams, with Asia-Pacific expected to account for over 30% of sales by 2030.
• Clinical Evidence: Demonstration of durable responses and favorable safety profiles will enhance clinician confidence, promoting adoption.

Projected Revenue

Market analysts estimate Jelmyto could generate USD 500 million annually globally by 2027, assuming steady market penetration and ongoing clinical expansion [4].

Potential Challenges

• Resistance from Conventional Therapies: Clinicians may prefer established intravesical agents unless Jelmyto demonstrates clear superiority.
• Pricing Pressures: Cost-containment measures may limit reimbursement, especially in public health systems.
• Regulatory Hurdles: As trials explore wider indications, delays or setbacks may limit market expansion.

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Strategic Recommendations

• Clinician Engagement: Educate urologists and oncologists through professional societies, emphasizing clinical trial data.
• Real-World Evidence (RWE): Collect and publish long-term data to validate Jelmyto’s efficacy and safety, supporting reimbursement negotiations.
• Alliance Development: Partner with healthcare providers and payers early to facilitate clinical integration.
• Pipeline Expansion: Support trials investigating combination therapies and broader indications to sustain growth momentum.

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Key Takeaways

• Jelmyto’s strategic clinical trial pipeline indicates potential for expanded indications and combination regimens, which could significantly increase its market footprint.
• The current bladder cancer therapeutics landscape favors minimally invasive, targeted approaches, positioning Jelmyto favorably if long-term efficacy is affirmed.
• Market barriers include high costs and clinicians' familiarity with traditional therapies; proactive engagement and real-world evidence are critical.
• Global regulatory momentum, especially in Europe and Asia-Pacific, offers avenues for rapid market expansion.
• Industry players must navigate reimbursement negotiations and competitive dynamics to capitalize on Jelmyto’s innovative potential.

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FAQs

1. What is the current approved indication for Jelmyto?
Jelmyto is FDA-approved for treating low-grade, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) in patients unwilling or unsuitable for cystectomy.

2. Are clinical trials ongoing to expand Jelmyto’s indications?
Yes. Trials are exploring Jelmyto's efficacy in high-grade papillary tumors and in combination with immunotherapies, aiming to broaden its therapeutic scope.

3. How does Jelmyto compare with traditional intravesical therapies?
Jelmyto offers targeted drug delivery directly into the bladder, with a non-invasive administration route. Early studies show comparable or superior response rates with a favorable safety profile, but long-term comparative data are still emerging.

4. What are the main market barriers facing Jelmyto?
High treatment costs, clinician familiarity with existing therapies, and variable reimbursement policies are primary obstacles. Overcoming these requires robust evidence and strategic payer engagement.

5. What is the projected market growth for Jelmyto over the next decade?
Estimated to reach USD 500 million annually globally by 2027, driven by indication expansion, increasing approvals, and rising bladder cancer incidence.

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Sources

1. Ljungberg B, et al. “FDA Approval of Jelmyto for Bladder Cancer: Clinical Data and Implications,” Urology Journal, 2023.
2. International Agency for Research on Cancer. “Bladder Cancer Fact Sheet,” 2022.
3. Grand View Research. “Bladder Cancer Therapeutics Market Size & Trends,” 2022.
4. MarketWatch. “Jelmyto Market Forecast and Industry Outlook,” 2022.

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This comprehensive analysis aims to provide business decision-makers with a detailed understanding of Jelmyto’s evolving clinical landscape, market potential, and strategic considerations.