FDA to host advisory committee meeting on UGN-102 for NMIBC

The FDA has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting for May 21, 2025, to discuss the submitted new drug application (NDA) for UGN-102 (mitomycin) for intravesical solution, which is currently under FDA review for recurrent low-grade, intermediate-risk non–muscle invasive bladder cancer (LG-IR-NMIBC).¹

The agency previously accepted the NDA for UGN-102 in October 2024, issuing a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025.²

The PDUFA target action date for UGN-102 is June 13, 2025.

UroGen explained in their news release, “The ODAC meeting will provide an opportunity for independent clinicians and other experts to evaluate the UGN-102 data and make a recommendation to the FDA as to whether the NDA should be approved and under what conditions.”

The company noted that although the FDA relies on the ODAC meeting to inform their pending regulatory decisions, the agency is not bound by the consensus that the group reaches.

“We are excited to discuss our data with the Advisory Committee and broader medical community as we continue our mission to bring innovative solutions to patients suffering from bladder cancer,” said Liz Barrett, President and CEO of UroGen, in the news release.¹ “We believe UGN-102 represents a meaningful advancement for patients facing the recurrent and challenging nature of LG-IR-NMIBC, and we look forward to the opportunity to discuss its potential.”

Data on UGN-102

The NDA for UGN-102 is primarily supported by findings from the phase 3 ENVISION trial (NCT05243550), which met its primary end point by demonstrating a 79.6% (95% CI, 73.9% to 84.5%) complete response rate in patients with LG-IR-NMIBC at 3 months following the first instillation of the therapy.

Updated data from the trial, which were reported in June 2024,³ showed that the 12-month duration of response (DOR) was 82.3% (95% CI, 75.9% to 87.1%) per Kaplan-Meier estimate among those patients who achieved a complete response at 3 months following the first instillation (n = 108) of UGN-102. The study also reported a DOR of 80.9% (95% CI, 73.9% to 86.2%) at both 15 (n = 43) and 18 (n = 9) months, per Kaplan-Meier estimates.

Regarding safety, the most common treatment-emergent adverse events (TEAEs) reported in the trial included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. According to UroGen, TEAEs were generally mild to moderate in severity. The safety profile for UGN-102 in the ENVISION trial was consistent with previous reports.

Overall, the single-arm, multinational, multicenter, ENVISION trial is evaluating the safety and efficacy of UGN-102 as a primary chemoablative therapy in patients with LG-IR-NMIBC. In total, the study enrolled 240 adult patients across 56 sites in the US and Europe.

Patients were eligible for enrollment in the trial if they had a negative voiding cytology for high-grade disease within 8 weeks before screening, adequate organ and bone marrow function, and an anticipated life expectancy of at least the duration of the trial.⁴ Those included in the study received 6 once-weekly intravesical instillations of UGN-102.

The primary end point for the study was the complete response rate at 3 months following the first instillation of the therapy. Secondary outcome measures include DOR, durable complete response rate, disease-free survival, and safety outcomes, all of which will be assessed for up to 63 months.

The ENVISION trial remains ongoing, with final study completion anticipated for 2028.

REFERENCES

1. UroGen announces FDA Advisory Committee for UGN-102, an investigational treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. News release. UroGen. May 7, 2025. Accessed May 7, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-fda-advisory-committee-ugn-102-investigational

2. UroGen announces FDA acceptance of its new drug application for UGN-102. News release. UroGen Pharma Ltd. October 15, 2024. Accessed May 7, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-fda-acceptance-its-new-drug-application-ugn-102

3. UroGen announces unprecedented 82.3% duration of response at 12 months in the ENVISION trial investigating UGN-102 as potentially the first FDA-approved non-surgical treatment for LG-IR-NMIBC. News release. June 13, 2024. Accessed May 7, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-unprecedented-823-duration-response-12-months

4. A phase 3 single-arm study of UGN-102 for treatment of low grade intermediate risk non-muscle-invasive bladder cancer (ENVISION). ClinicalTrials.gov. Last updated July 12, 2024. Accessed May 7, 2025. https://clinicaltrials.gov/study/NCT05243550