The U.S. Food and Drug Administration approved UroGen Pharma's Zusduri on Thursday, marking the first drug treatment for non-muscle invasive bladder cancer that has not spread beyond the inner layers of the organ. The approval provides patients with a non-surgical alternative to traditional procedures and sent the Israel-based company's U.S.-listed shares up approximately 50% in afternoon trading.
Clinical Trial Results Drive Approval
The FDA's decision was based on late-stage trial data involving 223 patients, in which 78% demonstrated a complete response to treatment, meaning all signs of cancer disappeared. This impressive efficacy rate convinced regulators to approve the drug despite concerns raised by an FDA advisory panel last month that narrowly opposed approval.
The advisory panel had criticized the single-arm study design, warning that such precedents could be problematic given existing surgical alternatives. However, the FDA ultimately moved forward with approval, appreciating the quality of the clinical trial results despite the lack of solid randomized data.
Addressing Unmet Medical Need
Non-muscle invasive bladder cancer affects approximately 82,000 people in the United States each year, with around 59,000 experiencing recurrence, according to UroGen. The disease primarily affects older populations, making less invasive treatment options particularly valuable for this patient demographic.
Current standard-of-care treatments include surgical procedures such as transurethral resection of bladder tumor, where doctors insert a thin tube through the urethra to remove the tumor. Zusduri offers an alternative approach through a gel-based formulation that keeps chemotherapy in the bladder for a longer duration, aiming to reduce tumor recurrence and avoid repeated surgeries.
Treatment Innovation and Availability
Scotiabank analyst George Farmer called the FDA decision a "major positive outcome," noting that regulators were "moved by the quality of the clinical trial results." The FDA appreciated the non-surgical treatment option, describing it as "a huge win for patients with recurrent low-grade tumors" as it involves "a simple drug instillation in a doctor's office."
UroGen expects Zusduri to be available in the United States on or around July 1, providing physicians and patients with this new therapeutic option for managing non-muscle invasive bladder cancer.
