The FDA has accepted UroGen Pharma's New Drug Application (NDA) for UGN-102 (mitomycin) intravesical solution for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). This decision marks a significant step toward potentially providing the first FDA-approved non-surgical treatment option for this patient population, with a Prescription Drug User Fee Act (PDUFA) goal date set for June 13, 2025.
UGN-102: A Novel Approach to NMIBC Treatment
UGN-102 is a formulation of mitomycin designed for intravesical administration, utilizing UroGen's RTGel technology. This technology allows for sustained release of the drug, prolonging exposure of the bladder tissue to mitomycin without the need for surgery. The drug is administered via a standard urinary catheter in an outpatient setting.
Liz Barrett, President and Chief Executive Officer of UroGen, stated, "The FDA acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients. UGN-102 could be the first FDA-approved medicine for LG-IR-NMIBC, offering a novel approach that could expand treatment options and address unmet needs."
ENVISION Trial: Supporting Data
The NDA is supported by data from the Phase 3 ENVISION trial (NCT05243550), a single-arm, multinational, multicenter study. The trial enrolled approximately 240 patients across 56 sites and evaluated the efficacy and safety of UGN-102 as primary chemoablative therapy.
The ENVISION trial met its primary endpoint, demonstrating a 79.6% complete response rate (CRR) at 3 months following the initial UGN-102 instillation. Furthermore, updated data showed an 82.3% 12-month duration of response (DOR) by Kaplan-Meier estimate among patients who achieved a complete response at 3 months.
Dr. Mark Schoenberg, Chief Medical Officer of UroGen, noted, "The NDA for UGN-102 is backed by a robust data set demonstrating impressive durability of response across three clinical trials and a favorable safety profile. Notably, the ENVISION trial successfully met its primary endpoint, showing a 79.6% complete response rate at three months after the first instillation of UGN-102. Additionally, the latest results from that trial revealed an 82.3% 12-month duration of response by Kaplan-Meier estimate in patients who achieved a complete response at 3 months."
The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The safety profile was consistent with prior studies, with TEAEs generally mild to moderate in severity.
Addressing an Unmet Need in Bladder Cancer
In the U.S., bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC accounts for approximately 22,000 new diagnoses each year and an estimated 60,000 recurrences annually. Current guidelines for NMIBC management include transurethral resection of bladder tumors (TURBT), but recurrence rates are high, with up to 70% of patients experiencing at least one recurrence.
"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," added Schoenberg.
