UroGen Pharma Submits FDA New Drug Application for UGN-102, a Potential Treatment for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

Image Credit: Adobe Stock Images/Adin

UroGen has completed its New Drug Application (NDA) submission for UGN-102 (mitomycin) for intravesical solution, suggesting that it has the potential to become the first FDA-approved treatment for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The NDA submission is supported by results from the Phase III ENVISION trial, which demonstrated that patients treated with UGN-102 had a 79.6% complete response (CR) rate at three months following the first instillation.¹

“The completion of the NDA submission for UGN-102 marks a crucial milestone for UroGen and underscores our dedication to advancing this groundbreaking treatment for patients with LG-IR-NMIBC,” said Liz Barrett, president, CEO, UroGen, in a press release. “By providing a viable alternative to repeated surgeries, if approved UGN-102 may offer patients quality of life benefits and clinically meaningful recurrence-free intervals. The high recurrence rates associated with LG-IR-NMIBC make the need for innovative therapies like UGN-102 urgent. UGN-102 could become a valuable new option for managing this challenging disease.”

ENVISION is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 for intravesical solution as chemoablative therapy in patients with LG-IR-NMIBC. The trial enrolled approximately 240 patients across 56 sites, with participants receving six once-weekly intravesical instillations of UGN-102. The primary endpoint of the study aimed to evaluate the CR rate at the three-month assessment after the first instillation, while the secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment.

Results also found that UGN-102 demonstrated an 82.3% 12-month duration of response (DOR) by Kaplan-Meier estimate (n=108) in patients who achieved a CR at three months. Further, DOR estimates at 15 (n=43) and 18 (n=9) months after 3-month CR were both 80.9%.

Common treatment-emergent adverse events (TEAEs) that resulted from the trial included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. In most cases, TEAEs were mild-to-moderate in severity and resolved or resolving. The safety profile of the ENVISION study remained consistent with previous studies of UGN-102.¹

According to the American Cancer Society, it is estimated that by the end of the year, 83,190 new cases of bladder cancer will be diagnosed in the United States, with a death rate of 16,840, a majority of which are expected to be male patients. Additionally, 9 out 10 people diagnosed with bladder cancer are over the age of 55 years old, with an average diagnosis age of 73 years old. In men, the chance of developing bladder cancer is 1 in 28, while women have a 1 in 89 chance.²

“About half of all bladder cancers are first found while the cancer is still only in the inner layer of the bladder wall,” reports the American Cancer Society. “These are non-invasive or in situ cancers. About 1 in 3 bladder cancers have spread into deeper layers of the bladder wall but are still only in the bladder. In most of the remaining cases, the cancer has spread to nearby tissues or lymph nodes outside the bladder. Rarely (in about 5% of cases), it has spread to distant parts of the body when it is first found.”

If the FDA accepts the NDA for UGN-102 and priority review in granted, UroGen anticipates FDA approval in early 2025. According to the company, this underscores its commitment to providing innovative treatment options for patients with high recurrence risks associated with LG-IR-NMIBC.¹

References

1. UroGen Submits Completed UGN-102 NDA Seeking Approval as the First FDA-Approved Treatment for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer. Business Wire. August 14, 2024. Accessed August 14, 2024. https://www.businesswire.com/news/home/20240814480495/en/UroGen-Submits-Completed-UGN-102-NDA-Seeking-Approval-as-the-First-FDA-Approved-Treatment-for-Low-Grade-Intermediate-Risk-Non-Muscle-Invasive-Bladder-Cancer

2. Key Statistics for Bladder Cancer. American Cancer Society. Accessed August 14, 2024. https://www.cancer.org/cancer/types/bladder-cancer/about/key-statistics.html