Despite a recent rebuff from an FDA advisory committee, the first treatment for a specific and underserved cohort of U.S. bladder cancer patients has crossed the FDA finish line as UroGen Pharma’s Zusduri.
The New Jersey-based pharma paired chemotherapy mitomycin with a sterile hydrogel using its sustained-release RTGel technology to create a localized therapy for potent tumor ablation of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). It’s a drug delivery combo familiar to UroGen from its 2020 approved Jelmyto, another hydrogel and mitomycin pairing formulated as a liquid solution made for delivery in the kidney’s pyelocalyceal system that is indicated for low-grade upper tract urothelial cancer.
In its phase 3 ENVISION trial, UroGen found that Zusduri could deliver a 78% complete response for patients with LG-IR-NMIBC at three months, with 79% of those patients still event-free 12 months later.
The FDA’s blessing is a “significant step forward” for the company and the 59,000 U.S. patients yearly who face the recurrent disease, CEO Liz Barrett said in UroGen’s Thursday release. Until now, patients with this type of bladder cancer have been limited to transurethral resection of bladder tumor, an anesthesia-requiring surgery that often must be repeated due to high recurrence rates.
“This historic achievement is a bold leap forward in our mission to redefine uro-oncology and bring innovation to patients who need it most,” Barrett added.
Zusduri is delivered directly into the bladder through an outpatient procedure using a urinary catheter to avoid the need for surgery. UroGen aims to make Zusduri available in the U.S. “on or around July 1.” However, the company still will have to complete its ongoing ENVISION trial as a post-marketing commitment with the FDA to “further characterize” the clinical benefit of the treatment.
Even so, the FDA’s backing is a twist from the outcome of a May meeting of its Oncologic Drugs Advisory Committee (ODAC). After reviewing the ENVISION study, the committee ultimately voted 4-5 against the drug’s benefit-risk profile, leaving UroGen “disappointed,” Barrett said at the time. The nearly split vote reflected a divide: some members were swayed by the drug’s non-surgical benefits for patients, while others were concerned about the single-arm trial design instead of a randomized study, OncLive reported.
UroGen chalked up $20.3 million in first-quarter Jelmyto sales, up 8% year-over-year, with “underlying demand” rising 12% in that same timeframe. The drug, UroGen’s only commercial product until Zusduri, is forecasted to bring in $94 million to $98 million in 2025 sales.