UroGen's New Drug Application for UGN-102 Accepted by FDA

UroGen Pharma Ltd. (Nasdaq: URGN), a biotechnology firm focused on developing innovative treatments for urothelial and specialty cancers, has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for UGN-102 (mitomycin) for intravesical solution. If approved, UGN-102 could become the first FDA-approved medication for treating low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The FDA's Prescription Drug User Fee Act (PDUFA) goal date for this application is set for June 13, 2025.

Liz Barrett, President and CEO of UroGen, highlighted the significance of this milestone, stating, "The FDA's acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients. If approved, UGN-102 will offer a novel approach to treatment, expanding options and addressing unmet needs in this area. We are committed to working with the FDA as we prepare for a potential launch in 2025."

Dr. Mark Schoenberg, Chief Medical Officer of UroGen, emphasized the strong data supporting the NDA, which includes results from three clinical trials. He noted that the ENVISION trial achieved its primary endpoint with a 79.6% complete response rate at three months post-treatment. Furthermore, 82.3% of patients who responded at three months maintained their response for 12 months, according to a Kaplan-Meier estimate. Common adverse effects in the trial included dysuria, hematuria, urinary tract infections, frequent urination, fatigue, and urinary retention. The safety profile observed in the ENVISION trial was consistent with previous studies of UGN-102. Dr. Schoenberg expressed confidence that, if approved, UGN-102 could significantly advance the management of LG-IR-NMIBC by achieving durable responses and potentially reducing recurrence rates.

UGN-102 is based on UroGen's proprietary RTGel® technology, a sustained-release hydrogel formulation designed to extend the exposure of bladder tissue to mitomycin, enabling non-surgical tumor treatment. Administered via a standard urinary catheter by healthcare professionals in an outpatient setting, UGN-102 is currently under regulatory review by the FDA, with a potential decision expected by mid-2025. The estimated U.S. market for LG-IR-NMIBC that UGN-102 could address is valued at around $5 billion.

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study assessing the efficacy and safety of UGN-102 as a primary chemoablative therapy in patients with LG-IR-NMIBC. The trial enrolled approximately 240 patients across 56 sites, with participants receiving six weekly intravesical instillations of UGN-102. The study's primary endpoint was the complete response rate at three months after the first instillation, and a key secondary endpoint was the durability of response over time in patients who achieved a complete response at three months.

Bladder cancer is the second most common urologic cancer in men in the U.S., with LG-IR-NMIBC accounting for 22,000 new diagnoses annually and around 60,000 recurrences each year among previously diagnosed patients. The median age of diagnosis is 73 years, and older patients often face increased risk of comorbidities. The standard of care for non-muscle invasive bladder cancer (NMIBC) typically includes transurethral resection of bladder tumor (TURBT), but recurrence rates are high, with up to 70% of NMIBC patients experiencing at least one recurrence.

UroGen Pharma Ltd., headquartered in Princeton, NJ, is dedicated to developing and commercializing novel treatments for urothelial and specialty cancers. The company has developed the RTGel® reverse-thermal hydrogel, a proprietary sustained-release platform technology aimed at improving the therapeutic profiles of existing drugs by enabling longer exposure of urinary tract tissue to medication. UroGen's first product targets low-grade upper tract urothelial cancer, and its investigational treatment UGN-102 aims to provide a non-surgical option for patients with LG-IR-NMIBC.