Dr Huang on the FDA Approval of Mitomycin Intravesical Solution for Recurrent, Low-Grade, Intermediate-Risk NMIBC

“The greatest implication of [this approval] is that, for the first time, we're going to have a completely novel way of treating patients with low-grade, intermediate-risk bladder cancer. It's going to be a paradigm shift for many patients from something that's primarily been surgical to something that is non-surgical and chemoablative.”

William C. Huang, MD, chief of the urology service at Tisch Hospital, NYU Langone Health, discussed the regulatory approval and clinical significance of the FDA approval of mitomycin intravesical solution (Zusduri; UGN-102) for the treatment of adult patients with recurrent, low-grade, intermediate-risk, non–muscle-invasive bladder cancer (NMIBC).

The approval was based on findings from the single-arm, multicenter phase 3 ENVISION trial (NCT05243550). Investigators enrolled 240 patients with low-grade NMIBC characterized by 1 to 2 intermediate-risk features, including multifocality, a solitary tumor larger than 3 cm, and/or recurrence within one year. PPrior transurethral resection of bladder tumor (TURBT) was also required. Mitomycin intravesical solution was administered at 7g mg weekly for 6 consecutive weeks. Tumor status was evaluated every 3 months by cystoscopy, urine cytology, and for-cause biopsy.

Among 223 patients evaluable for efficacy, the 3-month complete response (CR) rate was 78% (95% CI, 72%-83%), defined by absence of disease on cystoscopy and urine cytology, with biopsy confirmation as indicated. Duration of response (DOR) ranged from 0 to over 25 months, and the 12-month DOR rate was 79%.

Huang highlighted that the approval of mitomycin intravesical solution represents a significant shift in the management of low-grade, intermediate-risk NMIBC—offering a nonsurgical, chemoablative alternative to repeated TURBTs, which have long been the standard of care.

Huang noted that the approval was based not only on ENVISION, but also supported by data from prior studies such as the phase 2 OPTIMA II (NCT03558503) and phase 3 ATLAS (NCT04688931) trials, which demonstrated similar chemoablative efficacy with mitomycin intravesical solution.