FDA Clears NDA of Intravesical Mitomycin in Non–Muscle-Invasive Bladder Cancer

• The FDA has accepted the new drug application (NDA) of intravesical mitomycin (UGN-102) for the treatment of low-grade, intermediate-risk non–muscle-invasive bladder cancer (NMIBC).
• A Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025, has been set.
• If approved, this would mark the first agent to be FDA approved in this disease state.

The NDA of intravesical mitomycin as a treatment for low-grade, intermediate-risk NMIBC has been accepted by the FDA, and a PDUFA target action date of June 13, 2025, has been set.¹

"The FDA acceptance of our NDA is a pivotal moment in our journey to bring [intravesical mitomycin] to patients,” Liz Barrett, president and chief executive officer of UroGen, said in a press release. “[Intravesical mitomycin] could be the first FDA-approved medicine for [low-grade, intermediate-risk] NMIBC, 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 [intravesical mitomycin] in 2025.”

Intravesical mitomycin utilizes a sustained release hydrogel-based formulation to enable longer drug exposure to bladder tissue without surgery. The agent can be delivered by catheter in the outpatient setting.

The NDA is supported by the phase 3 ENVISION trial (NCT05243550), a study that enrolled an estimated 240 patients across 56 global sites. The primary end point was complete response rate (CRR), and secondary end points included duration of response, disease control rate, disease-free survival, and incidence of treatment-emergent adverse events.²

Previously reported data demonstrated a 79.6% CRR at 3-months following the initial treatment with intravesical mitomycin.¹ The safety profile was consistent with prior studies, and the agent was generally well tolerated.³

“The NDA for [intravesical mitomycin] 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 end point, showing a 79.6% complete response rate at 3 months after the first instillation of [intravesical mitomycin]. 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,” said Mark Schoenberg, MD, chief medical officer of UroGen, in a press release.¹

“We believe that, if approved, [intravesical mitomycin]’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] NMIBC,” added Schoenberg.

REFERENCES:

1. UroGen announces FDA acceptance of its new drug application for UGN-102. News release. UroGen Pharma Ltd. October 15, 2024. Accessed October 16, 2024. https://tinyurl.com/25xbhp98

2. A phase 3 single-arm study of UGN-102 for treatment of low grade intermediate risk non-muscle-invasive bladder cancer (ENVISION). ClinicalTrials.gov. Updated July 12, 2024. Accessed October 16, 2024. https://clinicaltrials.gov/study/NCT05243550

3. UGN-102, in development as the potential first non-surgical therapy for LG-IR-NMIBC, met primary endpoints in both phase 3 ATLAS and ENVISION clinical trials. News release. UroGen Pharma Ltd. July 28, 2023. Accessed October 16, 2024. https://tinyurl.com/yftt42pp