NDA Filed to FDA for Next-Gen Mitomycin UGN-103 in Recurrent NMIBC

A new drug application (NDA) has been submitted to the FDA for UGN-103 (mitomycin), an investigational intravesical solution for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).¹

The NDA submission is supported by data from the ongoing phase 3 UTOPIA trial (NCT06331299) of UGN-103 in 99 patients with recurrent LG-IR-NMIBC. In preliminary data announced in November 2025, UGN-103 produced a 3-month complete response (CR) rate of 77.8% (95% CI, 68.3%-85.5%). A more recent update announced in May 2026 showed durable disease control with UGN-103 among responders, with a 6-month estimated duration of response (DOR) of 94.5% (95% CI, 86.1%-97.9%).

UGN-103: Background and Clinical Development

UGN-103 is a next-generation formulation of mitomycin developed to overcome manufacturing and logistical constraints associated with preparation, handling, and storage of the currently approved therapy, UGN-102 (Zusduri). UGN-102 is a sustained-release, hydrogel-based intravesical solution already indicated for adults with recurrent LG-IR-NMIBC based on the phase 3 ENVISION trial (NCT05243550).

UGN-103 is built on UroGen’s proprietary RTGel platform, the same platform used for UGN-102. In April 2024, the FDA accepted the investigational new drug (IND) application for UGN-103, commencing its clinical development.

"UGN-103…is designed to provide a more streamlined manufacturing process, simplified reconstitution and extended shelf life of the reconstituted product while leveraging our RTGel technology,” said Liz Barrett, president and CEO of UroGen, in a news release.

Safety Profile

Safety findings from UTOPIA have been consistent with the established profile of UGN-102, with adverse events predominantly low grade.² The most commonly reported treatment-emergent adverse event, occurring in 10% of patients or more, was dysuria (19%). Serious adverse events occurred in 2% of patients, including atrial fibrillation, osteoarthritis, and respiratory failure, all of which were considered unrelated to treatment.

About the UTOPIA Trial

The phase 3 UTOPIA trial is a global, single-arm, multicenter study evaluating the efficacy and safety of UGN-103 in adult patients with LG NMIBC at intermediate risk of recurrence.³ Enrolled patients received 75 mg of UGN-103 by intravesical instillation in an outpatient setting once weekly for 6 weeks.

The primary end point is CR rate at 3 months, and patients who responded entered a follow-up period of up to 12 months to assess DOR. Secondary end points include duration of response, durable CR rate, incidence of treatment-emergent adverse events, and mitomycin plasma concentrations.

REFERENCES

1. UroGen Submits NDA for UGN-103, an Investigational Treatment of Recurrent LG-IR-NMIBC. News release. UroGen Pharma. August 17, 2026. Accessed August 17, 2026. https://tinyurl.com/2rvm34ce

2. Srivastava A, Vjaters E, Kaimakliotis HZ, et al. UTOPIA: A phase 3 trial of UGN-103, an intravesical mitomycin reverse thermal hydrogel, in recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. J Clin Oncol. 2026;44(16_suppl):4604-4604. doi:10.1200/jco.2026.44.16_suppl.4604

3. A phase 3 study of UGN-103 for treatment of patients with low-grade intermediate-risk non-muscle invasive bladder cancer (UTOPIA). ClinicalTrials.gov. Updated January 15, 2026. Accessed August 17, 2026. https://clinicaltrials.gov/study/NCT06331299