FDA receives NDA submission for UGN-103 in recurrent LG-IR-NMIBC

UroGen Pharma has submitted a new drug application (NDA) to the FDA seeking approval of UGN-103 (mitomycin) for intravesical solution for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).¹

UGN-103 is a next-generation mitomycin formulation that builds on the clinical and commercial foundation of Zusduri (mitomycin) for intravesical solution, which received FDA approval in June 2025.

"The NDA submission for UGN-103 marks another important milestone in advancing our vision to redefine the treatment of urothelial cancers," said Liz Barrett, president and CEO of UroGen, in the news release from the company.¹ "UGN-103 represents the next evolution in our portfolio and is designed to provide a more streamlined manufacturing process, simplified reconstitution, and extended shelf-life of the reconstituted product while leveraging our RTGel technology."

Data on UGN-103

The application is supported by data from the phase 3 UTOPIA trial (NCT06331299), which is an ongoing, single-arm, multicenter study evaluating the safety and efficacy of UGN-103 in adult patients with recurrent LG-IR-NMIBC.² The trial enrolled 99 patients, of whom 97% completed all 6 planned intravesical instillations. Patients received 75 mg of mitomycin once weekly for 6 weeks, with disease status assessed at 3 months using cystoscopy, for-cause biopsy, and urine cytology.

At the 3-month assessment, 77 of 99 patients achieved a CR, corresponding to a rate of 77.8% (95% CI, 68.3 to 85.5). Among patients without a CR, 16% had residual disease and 4% had progression to high-grade disease; 2% had missing or indeterminate responses. Among patients who achieved a CR at 3 months, the 6-month duration of response (DOR) was 94.5% by Kaplan-Meier estimate (95% CI, 86.1 to 97.9).

UroGen noted that these results are consistent with the 79.6% (95% CI, 73.9 to 84.5) CR rate and the 91.9% (95% CI, 86.9 to 95) 6-month DOR observed in the pivotal phase 3 ENVISION trial (NCT05243550), which led to the FDA approval of UGN-102 (mitomycin) for intravesical solution (Zusduri) in June 2025 for recurrent LG-IR-NMIBC.^3,4

Safety data on UGN-103 were based on the 99 patients who received at least 1 dose. Treatment-emergent adverse events (TEAEs) occurred in 60% of patients, with dysuria reported in 19%. Two patients experienced a total of 3 serious TEAEs—atrial fibrillation, osteoarthritis, and respiratory failure—and none were considered treatment related. No TEAE resulted in death.

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