Mitomycin Intravesical Solution Receives FDA Approval for Non-Muscle Invasive Bladder Cancer

Mitomycin intravesical solution (Zusduri, UroGen Pharma) was approved by the FDA for treatment of adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The decision is based on data from the ENVISION trial (NCT05243550) and makes mitomycin intravesical solution the first FDA-approved treatment for LG-IR-NMIBC.¹

Doctor holding bladder model | Image Credit: © M+Isolation+Photo - stock.adobe.com

Bladder cancer is the most common urologic cancers, of which NMIBC accounts for approximately 75% to 85% of all diagnoses. NIMBC is stratified based on risk. According to the International Bladder Cancer Group, intermediate risk NIMBC is defined as low-grade (LG) Ta tumor(s) that are multiple; large (over 3 cm in diameter); or recurrent within 1 year. The current standard of therapy for LG-IR-NMIBC—a persistent, frequently multifocal, recurrent cancer—is transurethral resection of bladder tumor (TURBT). However, this treatment approach is typically insufficient in controlling LG-IR-NMIBC.^2,3

UGN-102 is an innovative gel-based formulation of mitomycin created for the nonsurgical, primary treatment of LG-IR-NMIBC. It is delivered directly into the bladder via a urethral catheter in an outpatient setting, and begins as a cooled liquid before turning into a gel upon reaching body temperature. This transformation forms a temporary drug reservoir that remains in the bladder for up to 6 hours, gradually releasing mitomycin to ensure prolonged contact with tumor tissue while minimizing drug absorption into the bloodstream. Previous findings suggest that increasing mitomycin’s contact time in the bladder from 30 to 60 minutes after TURBT can significantly lower recurrence rates without raising the risk of systemic toxicity.²

In the single-arm, multicenter ENVISION trial, investigators assessed 240 adults with LG-IR-NMIBC that recurred after prior TURBT who received 75 mg mitomycin intravesical solution instilled once a week for 6 consecutive weeks. The primary end points were complete response (CR) at 3 months—defined as no detectable disease in the bladder by cystoscopy and urine cytology—and duration of response (DOR).³

The data showed that 78% of patients (95% CI, 72-83) achieved a complete response with a DOR ranging from 0 to over 25 months, and 79% of patients maintained a response for at least 12 months.³

The safety profile was favorable with the most common adverse effect (AE) being increased creatinine, increased potassium, dysuria, decreased hemoglobin, increased aspartate aminotransferase, increased alanine aminotransferase, increased eosinophils, decreased lymphocytes, urinary tract infection, decreased neutrophils, and hematuria. Twelve percent of patients experienced serious AEs including urinary retention (0.8%) and urethral stenosis (0.4%). A fatal adverse reaction of cardiac failure occurred in 1 patient.³

The FDA approval of mitomycin intravesical solution marks a significant advancement in the treatment of LG-IR-NMIBC, offering the first nonsurgical alternative for a patient population historically managed with TURBT alone. As recurrence remains a persistent challenge in this disease, the availability of a durable, outpatient therapy like UGN-102 represents a meaningful step forward in improving long-term outcomes and quality of life for patients.

"[Mitomycin intravesical solution] 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,” Sandip Prasad, MD, MPhil, director of Genitourinary Surgical Oncology at Morristown Medical Center/Atlantic Health System, NJ, and principal investigator of the ENVISION trial, said in a press release. “For decades, TURBT has been the standard approach for bladder cancer treatment. That’s why innovative treatments like [mitomycin intravesical solution] are essential, especially for those adult patients with recurrent low-grade, intermediate-risk NMIBC.”⁴

REFERENCES

1. A phase 3 single-arm study of UGN-102 for treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (ENVISION). Updated November 4, 2024. Accessed June 12, 2025. https://clinicaltrials.gov/study/NCT05243550

2. Deng S, Meng F, Wang L, et al. Global research trends in non-muscle invasive bladder cancer: Bibliometric and visualized analysis. Front Oncol. November 16, 2022. doi:10.3389/fonc.2022.1044830

3. Prasad SM, Shishkov D, Mihaylov NV, et al. Primary chemoablation of recurrent low-grade intermediate-risk nonmuscle-invasive bladder cancer with UGN-102: A single-arm, open-label, phase 3 trial (ENVISION). J Urol. February 1, 2025. doi:10.1097/JU.0000000000004296

4. FDA approves mitomycin intravesical solution for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. FDA. June 12, 2025. Accessed June 12, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mitomycin-intravesical-solution-recurrent-low-grade-intermediate-risk-non-muscle

5. 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). Business Wire. June 12, 2025. Accessed June 12, 2025. https://www.businesswire.com/news/home/20250610841284/en/U.S.-FDA-Approves-UroGens-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