UGN-103, a next-generation mitomycin formulation built on the ZUSDURI foundation, is supported by three-month CR and six-month DOR data from the phase 3 UTOPIA trial.
A new drug application (NDA) has been submitted to the FDA for UGN-103 (mitomycin) for intravesical solution, an investigational next-generation formulation for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).1
The submission is supported by data from the ongoing phase 3 UTOPIA trial (NCT06331299), a single-arm, multicenter study evaluating UGN-103 in this population.¹,² UGN-103 produced a three-month complete response (CR) rate of 77.8% (95% CI, 68.3%-85.5%) and a six-month duration of response (DOR) rate of 94.5% by Kaplan-Meier estimate (95% CI, 86.1%-97.9%).
"The NDA submission for UGN-103 marks another important milestone in advancing our vision to redefine the treatment of urothelial cancers," Liz Barrett, president and chief executive officer of UroGen, said in a news release.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."
UGN-103 is designed to offer key improvements compared with mitomycin for intravesical solution (Zusduri; UGN-102), which is the first and only FDA-approved treatment for the treatment of adult patients with low-grade, intermediate-risk NMIBC. The notable improvements include a shorter manufacturing process and a simplified reconstitution procedure without compromising prolonged drug exposure within the bladder.
In June 2025,
UTOPIA is a single-arm, multicenter study that enrolled patients with low-grade intermediate-risk NMIBC with an anticipated life expectancy of at least the duration of the study.1,2 Other key inclusion criteria included a history of at least 1 prior episode of NMIBC, negative voiding cytology for high-grade disease within 8 weeks prior to screening, and adequate organ and bone marrow function. Intermediate-risk disease was defined as having 1 or 2 of the following characteristics: presence of multiple tumors; solitary tumor over 3 cm in diameter; and/or early or frequent recurrence.
Following enrollment, all patients received UGN-103 at 75 mg once weekly for 6 weeks for a total of 6 doses. The primary end point was 3-month CR rate. Secondary end points included DOR, durable CR rate, safety, mitomycin plasma concentrations, and mitomycin maximum plasma concentration.UTOPIA enrolled 99 patients with recurrent LG-IR-NMIBC across global sites.¹
