PDUFA Goal Date Set for June 13, 2025: UGN-102 Could Be First FDA-Approved Treatment for LG-IR-NMIBC
UroGen Pharma Ltd. (Nasdaq: URGN), a biotechnology company committed to developing innovative solutions 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 would become the first FDA-approved medication for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The FDA has established a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025.
“The FDA’s acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients,” said Liz Barrett, President and CEO of UroGen. “If approved, UGN-102 could offer a novel treatment option that expands choices and addresses unmet medical needs in this area. We recognize the urgent demand for innovative therapies and are committed to collaborating with the FDA in preparation for a potential launch in 2025.”
Dr. Mark Schoenberg, Chief Medical Officer of UroGen, added, “Our NDA for UGN-102 is supported by robust data demonstrating significant durability of response across three clinical trials, along with a favorable safety profile. Notably, the ENVISION trial achieved its primary endpoint, showing a 79.6% complete response rate three months after the initial UGN-102 instillation. Furthermore, recent data indicated an 82.3% 12-month duration of response in patients who achieved a complete response at three months. The most frequently reported treatment-emergent adverse events included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The safety profile observed in the ENVISION trial aligns with findings from other studies involving UGN-102. We believe that, if approved, UGN-102 could significantly advance the management of LG-IR-NMIBC by achieving durable complete responses and potentially reducing recurrence rates while extending treatment-free intervals.”
About UGN-102
UGN-102 (mitomycin) for intravesical solution is an innovative formulation currently under regulatory review for the treatment of LG-IR-NMIBC. Leveraging UroGen’s proprietary RTGel® technology, UGN-102 features a sustained-release, hydrogel-based formulation designed to maximize exposure of bladder tissue to mitomycin, allowing for non-surgical tumor treatment. UGN-102 is administered to patients through a standard urinary catheter in an outpatient setting by a trained healthcare professional. The NDA for UGN-102 is currently under review by the FDA, with a potential decision expected by June 13, 2025. If approved, UGN-102 could tap into a U.S. market for LG-IR-NMIBC estimated at approximately $5 billion.
About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational study assessing the efficacy and safety of UGN-102 (mitomycin) as a primary chemoablative therapy for patients with LG-IR-NMIBC. The trial successfully enrolled approximately 240 patients across 56 sites, with participants receiving six once-weekly intravesical instillations of UGN-102. The primary endpoint assessed the complete response rate at the three-month mark after the first instillation, while the key secondary endpoint measured the durability of response in patients who achieved a complete response at three months. For more details on the ENVISION trial, visit www.clinicaltrials.gov (NCT05243550).
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Bladder cancer is the second most common urologic cancer among men in the U.S. LG-IR-NMIBC accounts for approximately 22,000 new diagnoses each year and an estimated 60,000 recurrences among previously diagnosed patients. This cancer predominantly affects older individuals, with a median diagnosis age of 73 and a heightened risk of comorbidities. Standard care guidelines for managing NMIBC recommend transurethral resection of bladder tumor (TURBT). Up to 70% of NMIBC patients experience at least one recurrence, and those with LG-IR-NMIBC are even more likely to face recurrence, often necessitating repeated TURBT procedures.
About UroGen Pharma Ltd.
UroGen Pharma Ltd. is a biotechnology company focused on developing and commercializing innovative therapies for urothelial and specialty cancers, aiming to provide patients with improved treatment options. UroGen has created RTGel® reverse-thermal hydrogel, a proprietary sustained-release technology that enhances the therapeutic profiles of existing drugs. This sustained-release approach allows for prolonged exposure of urinary tract tissue to medications, potentially improving the efficacy of local therapies. UroGen’s first approved product treats low-grade upper tract urothelial cancer, and UGN-102 (mitomycin) for LG-IR-NMIBC is designed for non-surgical tumor ablation. Headquartered in Princeton, NJ, UroGen also has operations in Israel. For more information, visit www.UroGen.com or follow UroGen on X (formerly Twitter) at @UroGenPharma.
