FDA Approves UroGen Pharma’s Zusduri for Intravesical Solution in Recurrent Low-Grade, Intermediate-Risk Non-Muscle Invasive Bladder Cancer

The FDA has approved UroGen Pharma’s Zusduri (mitomycin) for intravesical solution, making it the first and only FDA-approved treatment specifically indicated for adults with recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). According to the company, the treatment enables localized, prolonged delivery of mitomycin for targeted tumor ablation through the use of its RTGel sustained-release hydrogel technology. Approval was based on results from the Phase III ENVISION trial, which demonstrated a high complete response (CR) rate.¹

How Does Zusduri Change the Treatment Landscape for Recurrent LG-IR-NMIBC?

“The approval of Zusduri represents a significant step forward for our company and for the treatment of recurrent LG-IR-NMIBC," said Liz Barrett, president, CEO, UroGen, in a press release. "For the first time, the estimated 59,000 US patients facing recurrent LG-IR-NMIBC each year have access to an FDA-approved medicine. This historic achievement is a bold leap forward in our mission to redefine uro-oncology and bring innovation to patients who need it most. We are deeply grateful to the FDA for their collaboration and to the investigators, patients, and caregivers whose commitment made this milestone possible. Their contributions have been essential in bringing meaningful innovation to the bladder cancer community.”

Trial Design and Endpoints

• The single-arm, multinational, multicenter ENVISION trial evaluated the safety and efficacy of Zusduri for intravesical solution as chemoablative therapy in 240 patients with LG-IR-NMIBC.
• All patients received six once-weekly intravesical instillations.
• The primary endpoint of the study was the CR rate at the three-month assessment after the first instillation, while the secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment.²

Efficacy and Safety Data

• Results show that Zusduri demonstrated a 78% CR at three months, with 9% of responders remaining event-free after one year.1
• Additionally, the treatment demonstrated an 82.3% 12-month duration of response (DOR) in patients who achieved a CR at three months.
• DOR estimates at 15 and 18 months after 3-month CR were both 80.9%.²
• Common adverse events (AEs) associated with Zurduri included creatinine; increased potassium; dysuria; decreased hemoglobin; increased aspartate aminotransferase; increased alanine aminotransferase; increased eosinophils; decreased lymphocytes; urinary tract infection; decreased neutrophils; and hematuria.
• Serious AEs were reported in 12% of patients, including urinary retention and urethral stenosis.¹

Regulatory and Epidemiological Context

The approval comes despite the FDA’s Oncologic Drugs Advisory Committee (ODAC) narrowly voting against the application for Zusduri. Last month, ODAC voted four-to-five against the benefit-risk profile of the medication.³

“While we are disappointed by today’s outcome, we continue to believe our clinical data support UGN-102 for the treatment of recurrent LG-IR-NMIBC, a disease with no FDA-approved therapies,” Barrett said in a prior press release regarding the decision. “The FDA carefully considers the independent advice from ODAC, and we look forward to working with the FDA as they complete their review of the application for UGN-102.”³

According to the American Cancer Society, bladder cancer is more common in men than it is women in the United States. Currently, it is the 10th leading cause of cancer-related deaths in the United States. Men maintain a 0.8% chance of dying from the disease, while women have a 0.3% chance. However, rates have been slowly declining over the past few decades, with an estimated 1% decline per year since 2013. By the end of this year, it is estimated that there will be 84,870 new cases of bladder cancer in the United States, with 17,420 deaths.⁴

"Zusduri marks a breakthrough in uro-oncology, offering a new alternative for recurrent LG-IR-NMIBC patients who can live for many years with the disease but often endure multiple resections, under general anesthesia,” ENVISION trial principal investigator Sandip Prasad, MD, MPhil, director, genitourinary surgical oncology, Morristown Medical Center/Atlantic Health System, NJ, said in the press release. “For decades, TURBT has been the standard approach for bladder cancer treatment. That’s why innovative treatments like Zusduri are essential, especially for those adult patients with recurrent low-grade, intermediate-risk NMIBC.”

References

1. U.S. FDA Approves UroGen’s ZUSDURI™ (mitomycin) for Intravesical Solution as the First and Only Medication for Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC). UroGen. June 12, 2025. Accessed June 13, 2025. https://investors.urogen.com/news-releases/news-release-details/us-fda-approves-urogens-zusduritm-mitomycin-intravesical

2. UroGen Pharma Submits FDA New Drug Application for UGN-102, a Potential Treatment for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer. PharmExec. August 14, 2024. Accessed June 13, 2025. https://www.pharmexec.com/view/urogen-pharma-fda-new-drug-application-ugn-102-potential-treatment-low-grade-intermediate-risk-non-muscle-invasive-bladder-cancer

3. UroGen Announces Outcome of Oncologic Drugs Advisory Committee for UGN-102 for the Treatment of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC). UroGen. May 21, 2025. Accessed June 13, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-outcome-oncologic-drugs-advisory-committee-ugn

4. Key Statistics for Bladder Cancer. American Cancer Society. Accessed June 13, 2025. https://www.cancer.org/cancer/types/bladder-cancer/about/key-statistics.html