The FDA accepted an NDA for UGN-103 for recurrent low-grade intermediate-risk NMIBC, with a target action date of June 14, 2027.
The FDA has accepted a new drug application (NDA) seeking the approval of UGN-103 (mitomycin) for intravesical solution for the treatment of adult patients with recurrent low-grade intermediate-risk non–muscle-invasive bladder cancer (NMIBC); the regulatory agency assigned a Prescription Drug User Fee Act target action date of June 14, 2027.¹
The application is supported by data from the single-arm, phase 3 UTOPIA trial (NCT06331299), which demonstrated a complete response (CR) rate of 77.8% (95% CI, 68.3%-85.5%) at 3 months.1,2 Among patients achieving CR, the Kaplan-Meier estimated probability of remaining event-free at 6 months was 94.5%.¹
“FDA acceptance of the UGN-103 NDA advances our vision for the next evolution of our portfolio and our continued commitment to transforming the treatment of recurrent LG-IR-NMIBC,” Liz Barrett, president and chief executive officer of UroGen Pharma, stated in the news release.¹ “Building on our experience with [UGN-102 (Zusduri)] for intravesical solution, UGN-103 combines our proprietary RTGel technology with a novel mitomycin formulation, which leverages a streamlined manufacturing process and simplified reconstitution.”
In June 2025,
UGN-103 incorporates mitomycin into a thermally responsive hydrogel.1 The formulation is liquid under chilled conditions, allowing intravesical administration, and converts into a semisolid gel depot after instillation into the bladder. This delivery system is designed to sustain local mitomycin exposure.
UTOPIA was an open-label, single-arm, multicenter trial that enrolled 99 patients at least 18 years of age with histologically confirmed low-grade Ta NMIBC, with at least 1 previous NMIBC episode.4 Intermediate-risk disease required 1 or 2 of the following features: multiple tumors, a solitary tumor larger than 3 cm, or early or frequent recurrence, defined as at least 1 occurrence of low-grade NMIBC within 1 year of the current diagnosis.
Additional requirements included negative voiding cytology for high-grade disease within 8 weeks before screening and adequate organ and bone marrow function. Renal eligibility required an estimated glomerular filtration rate of at least 30 mL/min.²
Patients were excluded if they received BCG treatment within the previous year, had high-grade bladder cancer within 2 years, had current T1 disease, or had past or current muscle-invasive bladder cancer. Prior intravesical chemotherapy within 2 years also excluded patients from enrollment, except for a single immediate postoperative dose after transurethral resection of bladder tumors.
Enrolled patients received UGN-103 at 75 mg by intravesical instillation once weekly for 6 weeks. The primary end point was CR rate at the 3-month visit, approximately 3 months after the first instillation.
Registry results, posted September 28, 2026, summarized data through March 18, 2026. Among 99 treated patients, 59.6% experienced treatment-emergent adverse effects (TEAEs). Two patients (2.02%) experienced serious TE AEs, and 37.4% experienced TE AEs of special interest. AEs did not lead to death in any patients.
The safety results for UGN-103 were consistent with data for UGN-201 from ENVISION.1
