FDA panel split on UroGen’s bladder cancer drug

PRINCETON, N.J. - UroGen Pharma Ltd. (NASDAQ: URGN), a biotech firm focusing on urothelial and specialty cancers with a market capitalization of $348 million, faced a divided response from the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) regarding its new drug application for UGN-102 (mitomycin) for intravesical solution. The committee’s vote on Monday concluded with a narrow 4 to 5 against the drug’s favorable benefit/risk profile for treating recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The news triggered a sharp 23.6% decline in the stock price over the past week, according to InvestingPro data.

Despite the setback, UroGen’s President and CEO Liz Barrett remains optimistic, stating their commitment to working with the FDA to review the application further. The FDA typically takes ODAC recommendations into account, but is not bound by them. InvestingPro analysis shows the company maintains strong financial flexibility with a healthy current ratio of 5.65 and more cash than debt on its balance sheet, potentially providing runway for continued development efforts.

UGN-102 is designed to be a non-surgical treatment for LG-IR-NMIBC, a condition currently lacking FDA-approved therapies. The drug utilizes UroGen’s proprietary RTGel technology, intended to allow prolonged exposure of bladder tissue to mitomycin. The Phase 3 ENVISION trial’s results, which evaluated the drug’s efficacy and safety, were part of the data considered by ODAC.

The most common adverse events reported in the ENVISION trial included dysuria, hematuria, and urinary tract infections, aligning with the safety profile observed in other UGN-102 studies. The FDA’s review of UGN-102 is ongoing, with a decision expected by the Prescription Drug User Fee Act (PDUFA) date of June 13, 2025.

LG-IR-NMIBC affects approximately 82,000 Americans annually, with a high recurrence rate, especially among the elderly who may face repeated surgeries. UroGen Pharma’s mission is to provide better treatment options for such diseases, leveraging its sustained-release technology to potentially enhance existing drug therapies. The company demonstrates impressive gross profit margins of nearly 90%, though analysts currently don’t anticipate profitability this year. InvestingPro analysis suggests the stock is currently undervalued, with multiple additional insights available in the comprehensive Pro Research Report, which provides deep-dive analysis of this and 1,400+ other US stocks.

This article is based on a press release statement and aims to present the facts surrounding UroGen Pharma’s recent developments without promotional content. The information provided herein is subject to risks and uncertainties, and actual results may differ from those discussed.

In other recent news, UroGen Pharma reported its Q1 2025 earnings, revealing a revenue shortfall with earnings per share (EPS) of -$0.92, missing the forecast of -$0.79. Revenue for the quarter was $20.25 million, below the expected $22.5 million. This financial miss comes as UroGen Pharma prepares for the potential launch of UGN-102 in July 2025. Concerns have arisen after FDA staff questioned the efficacy of UGN-102, a bladder cancer drug, particularly its complete response rate compared to the current standard of care. Despite these concerns, Oppenheimer maintained an Outperform rating with a $36 price target, while TD Cowen expressed confidence in a positive advisory panel vote. The FDA’s briefing documents highlighted the need for a randomized trial design, contrasting with UroGen’s single-arm pivotal trial approach. UroGen is actively preparing for an upcoming advisory committee meeting, which could significantly impact the future of UGN-102.

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