NDA Granted to Investigational Mitomycin Formulation

The US Food and Drug Administration has accepted a New Drug Application for the investigational drug UGN-102 (mitomycin) from UroGen Pharma as an intravesical therapy. UGN-102 could become the first FDA-approved therapy for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The mechanism of action for UGN-102 involves a sustained-released hydrogel formulation of mitomycin that allows for longer exposure of bladder tissue to the drug, potentially sparing patients from surgical intervention.

UGN-102 was studied in the phase 3, single-arm, multinational, multicenter ENVISION trial as a primary chemoablative therapy for LG-IR-NMIBC. The trial evaluating efficacy and safety enrolled approximately 240 patients who received six once-weekly intravesical instillations of the mitomycin formula. The primary endpoint was complete response (CR) rate at three months after instillation; the key secondary endpoint evaluated durability over time in patients who did achieve CR at their three-month assessment.

The trial met its primary endpoint with a 79.6% CR rate at three months, with later results from the trial revealing an 82.3% 12-month duration of response according to Kaplan-Meier estimates.

Dr. Mark Schoenberg, CMO of UroGen, noted that “The NDA for UGN-102 is backed by a robust data set demonstrating impressive durability of response,” and if approved, will be “a significant advance in managing LG-IR-NMIBC.”