UroGen Pharma Ltd. announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for UGN-102 (mitomycin) for intravesical solution. This investigational drug is intended for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). If approved, UGN-102 could become the first FDA-approved medicine for this specific indication, offering a novel non-surgical approach. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025.
Clinical Trial Data and Efficacy
The NDA for UGN-102 is supported by data from three clinical trials, including the Phase 3 ENVISION trial. The ENVISION trial, a single-arm, multinational, multicenter study, evaluated the efficacy and safety of UGN-102 as primary chemoablative therapy in patients with LG-IR-NMIBC. The trial met its primary endpoint, demonstrating a 79.6% complete response rate at three months following the first instillation of UGN-102. Furthermore, recent data from the ENVISION trial showed an 82.3% 12-month duration of response by Kaplan-Meier estimate in patients who achieved a complete response at 3 months.
Safety Profile
The safety profile of UGN-102, as observed in the ENVISION trial, was consistent with previous studies. The most common treatment-emergent adverse events included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention.
Mechanism of Action and RTGel Technology
UGN-102 is a formulation of mitomycin utilizing UroGen’s proprietary RTGel technology. RTGel is a sustained-release, hydrogel-based platform designed to prolong the exposure of bladder tissue to mitomycin. This technology enables the non-surgical treatment of tumors through intravesical administration via a standard urinary catheter in an outpatient setting.
Market Opportunity and Disease Burden
LG-IR-NMIBC affects approximately 22,000 newly diagnosed patients each year in the U.S., with an estimated 60,000 recurrences annually. Current guidelines recommend transurethral resection of bladder tumor (TURBT) as the standard of care. However, up to 70% of NMIBC patients experience recurrence, necessitating repeated TURBT procedures. The U.S. market for LG-IR-NMIBC is estimated at approximately $5 billion, representing a significant opportunity for UGN-102 if approved.
UroGen's Perspective
"The FDA acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients," said Liz Barrett, President and Chief Executive Officer of UroGen. Dr. Mark Schoenberg, Chief Medical Officer of UroGen, added, "We believe that, if approved, UGN-102’s ability to achieve durable complete responses and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC."
