The FDA has approved UroGen Pharma’s mitomycin intravesical solution, now marketed as Zusduri, for the treatment of adult patients with recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
The decision marks a significant milestone as Zusduri becomes the first FDA-approved non-surgical, chemoablative therapy for this specific bladder cancer population.
Zusduri is a unique formulation of the chemotherapy mitomycin, delivered using a reverse thermal sterile sustained-release RTGel hydrogel technology.
The solution is instilled into the bladder via a urinary catheter as a liquid and turns into a gel at body temperature, enabling prolonged drug exposure to the bladder lining. The delivery system allows for enhanced local activity of the chemotherapy agent while minimizing systemic absorption to achieve effective local tumor ablation. It is performed as an outpatient procedure as it does not involve surgery.
Despite a recent setback from an FDA advisory committee, Zusduri was able to secure the FDA approval to make it the first treatment specifically approved for a previously underserved group of bladder cancer patients.
The current standard of care for LG-IR-NMIBC is transurethral resection of bladder tumor (TURBT), a surgical procedure usually performed under general anesthesia. Because of the high recurrence rates associated with this cancer type, patients often require repeat TURBT procedures over time.
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Zusduri’s chemoablative approach provides a non-surgical option that could significantly reduce the treatment burden for patients who experience frequent recurrences.
The drug delivery approach mirrors UroGen’s Jelmyto, a hydrogel-mitomycin combination formulated as a liquid for delivery into the kidney’s pyelocalyceal system to treat low-grade upper tract urothelial cancer. Jelmyto received FDA approval in 2020.
“The approval of Zusduri represents a significant step forward for our company and for the treatment of recurrent LG-IR-NMIBC,” said Liz Barrett, president and CEO of UroGen, in a news release from the company announcing the approval.
“For the first time, the estimated 59,000 US patients facing recurrent LG-IR-NMIBC each year have access to an FDA-approved medicine. This historic achievement is a bold leap forward in our mission to redefine uro-oncology and bring innovation to patients who need it most.”
Zusduri’s approval is based on positive results from the Phase III Envision trial, a multicenter, single-arm study evaluating the efficacy and safety of six weekly instillations of Zusduri in adults with recurrent LG-IR-NMIBC.
The trial enrolled 240 patients, with 223 evaluable for the primary efficacy analysis.
At three months post-treatment, 78% of patients achieved a complete response. Furthermore, 79% of responders remained in complete response for at least 12 months, and longer-term data showed sustained benefit for many patients beyond 18 months.
Despite the promising efficacy data, the FDA’s decision came after a narrow 5-4 vote against approval by the Oncologic Drugs Advisory Committee (ODAC) last month. Committee members raised concerns about the lack of a randomized control arm and limited long-term follow-up.
However, the FDA ultimately determined that the drug’s benefits outweighed these limitations, with some members seemingly won over by the non-surgical benefit.
As part of its post-marketing obligations to the FDA, UroGen has agreed to complete the ongoing Envision trial to further assess the clinical benefit of Zusduri in patients with recurrent LG-IR-NMIBC.
Additionally, UroGen will provide the FDA with annual updates on the duration of response (DOR) for all patients maintaining complete responses. These updates will continue until each patient either experiences a recurrence, disease progression, death, is lost to follow-up or reaches 63 months after the first instillation — whichever comes first, in accordance with the study protocol.
UroGen said Zusduri is expected to be available in the US on or around July 1, 2025, for the treatment of adults with recurrent LG-IR-NMIBC.
UroGen reported $20.3 million in Jelmyto sales for the first quarter, marking an 8% increase year-over-year, with underlying demand climbing 12% during the same period. Jelmyto, the company’s sole commercial product before Zusduri’s approval, is projected to generate between $94 million and $98 million in sales for 2025.
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