FDA Accepts New Drug Application for UGN-102 in Bladder Cancer

A new drug application (NDA) has been accepted by the Food and Drug Administration (FDA) for the intravesical solution of UGN-102 (mitomycin) in the treatment of low-grade intermediate-risk non-muscle-invasive bladder cancer, positioning UGN-102 to potentially become the first FDA-approved agent for this patient population, according to a press release from UroGen Pharma, the drug’s manufacturer. Notably, the Prescription Drug User Fee Act (PDUFA) goal date for the application is June 13, 2025.

“The FDA acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients,” Liz Barrett, president and chief executive officer of UroGen stated in the press release. “UGN-102 could be the first FDA-approved medicine for [low-grade intermediate-risk non–muscle-invasive bladder cancer], offering a novel approach that could expand treatment options and address unmet needs. There is an urgent need for innovative solutions in this space, and we are dedicated to collaborating with the FDA as we prepare for a potential launch of UGN-102 in 2025.”

UGN-102 for intravesical solution may potentially serve as a unique treatment option, as the agent utilizes an innovative drug formulation of mitomycin. UGN-102 is a sustained-release, hydrogel-based formulation developed to prolong bladder tissue exposure to mitomycin through the use of UroGen’s proprietary RTGel technology. In turn, this allows for the treatment of tumors without surgery. Additionally, UGN-102 is administered to patients through a standard urinary catheter in an outpatient setting by a trained healthcare professional.

The application acceptance is supported by data from the phase 3 ENVISION trial. The single-arm, multinational, multicenter trial is evaluating the safety and efficacy of the investigative treatment for intravesical solution as primary chemoablative therapy in patients with low-grade intermediate-risk non-muscle-invasive bladder cancer. The release states that ENVISION has completed the target enrollment of approximately 240 patients across 56 treatment sites.

Eligible patients enrolled onto the trial were treated with six once-weekly intravesical instillations of UGN-102. The primary end point of the study was complete response (CR; the disappearance of cancer) rate at the three-month assessment following the first instillation and key secondary end points evaluated durability over time in participants who achieved a CR at the three-month assessment.

“The ENVISION trial successfully met its primary end point, showing a 79.6% [CR] 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 three months,” Dr. Mark Schoenberg, chief medical officer of UroGen, stated in the release. “The most common treatment-emergent side effects in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. Additionally, the safety profile observed in the ENVISION trial was consistent with that seen in other studies of UGN-102.”

According to the release from UroGen, bladder cancer is the second most common urologic cancer among men in the United States. This disease represents approximately 22,000 new diagnoses annually and an estimated 60,000 recurrences each year among patients previously diagnosed. Notably, bladder cancer typically affects patients among the older population who have a higher risk of comorbidities, and has a median diagnosis age of 73.

Current treatment guidelines for this disease typically compromise transurethral resection of bladder tumor as the standard of care. However, nearly 70% of patients with bladder cancer experience at least one recurrence. Patients with non-muscle-invasive bladder cancer are at an even higher risk and are more likely to recur, leading to repeated procedures.

“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. … 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 low-grade intermediate-risk non–muscle-invasive bladder cancer,” Schoenberg said.

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