UroGen Says Bladder Cancer Treatment Accepted for Review, With Decision Due in June 2027
Key Facts
UroGen Pharma said the U.S. Food and Drug Administration had accepted a new drug application for its experimental bladder cancer treatment UGN-103, a regulatory step that paves the way for review of its marketing application. The application covers adults with recurrent low-grade, intermediate-risk non-muscle-invasive bladder cancer, according to the company's statement. The candidate is mitomycin administered into the bladder, and its development is part of UroGen's efforts to improve manufacturing and preparation compared with its treatment Zusduri. The company said the candidate is designed to offer "a shorter manufacturing process and a simplified reconstitution procedure." The announcement concerns acceptance of the drug application for review, a stage preceding a regulatory decision on whether to allow it to be marketed.
In clinical results announced by UroGen, the treatment candidate achieved a complete response rate of 77.8% after 3 months in the UTOPIA trial. The company's annual report described the trial as single-arm and multicenter, a design that follows a group receiving the treatment candidate. Enrollment of 99 patients was completed across multiple centers globally, according to the same report. Participants received mitomycin administered into the bladder at a dose of 75 mg each time. The treatment protocol called for dosing once weekly for 6 weeks, while the complete response result was reported at the third-month assessment.
For comparison, the company reported a complete response rate of 79.6% after 3 months for Zusduri in the separate ENVISION trial. In follow-up of complete responders in UTOPIA, the estimated probability of remaining in response at 6 months was 94.5%. Response durability in this group was calculated using the Kaplan–Meier method, according to UroGen's statement on the follow-up results. The estimated probability of remaining in response with Zusduri at 6 months was 91.9% in the ENVISION trial. These results relate to groups in separate trials and are not a direct comparison between the treatment candidate and the approved treatment within a single trial.
The U.S. Food and Drug Administration previously approved UroGen's mitomycin for administration into the bladder under the name Zusduri on June 12, 2025. In the treatment context for this disease, the U.S. National Cancer Institute says treatment for intermediate-risk bladder cancer typically includes transurethral tumor resection and fulguration. This is followed by treatment administered into the bladder using the BCG vaccine or chemotherapy with mitomycin or gemcitabine, according to the institute. UroGen explains that its thermal gel technology is liquid at low temperatures. It turns into a gel at body temperature, according to the company's description of its properties.
The treatment candidate's regulatory progress comes alongside growth in sales of Zusduri, which generated revenue of $50.4 million in the second quarter of 2026. UroGen said in its financial results that this represented growth of 73% from the previous quarter. The company also reported that 1,444 sites had been activated to provide care with Zusduri as part of its second-quarter results. On the approved treatment's safety, the Food and Drug Administration recorded serious adverse reactions in 12% of patients in its original approval data. This percentage relates to Zusduri, which received the earlier approval, and does not represent a safety result for the treatment candidate UGN-103.
The development collaboration for the treatment candidate with medac dates back to January 2024, when UroGen entered into an agreement with the company. The agreement covered licensing and supply, according to UroGen's statement announcing acceptance of the new drug application. The company expects intellectual property protection for the treatment candidate to extend to July 2044. On the clinical front, the UTOPIA protocol includes a follow-up phase of up to 12 months for complete responders to assess response durability. For the next regulatory milestone, the Food and Drug Administration has set a target decision date of June 14, 2027, for the treatment application, according to the company's statement.