UroGen Stock Drops 47% After FDA Panel Rejects Bladder Cancer Drug

UroGen Pharmaceuticals (URGN.US) experienced a significant setback on Wednesday as its stock price plummeted by approximately 47%. This dramatic decline followed the decision by the U.S. Food and Drug Administration (FDA) advisory committee to vote against the company's bladder cancer treatment, UGN-102 (mitomycin). The drug, currently under regulatory review in the United States, was evaluated for its risk-benefit profile in the context of intravesical injection solutions.

The FDA's Oncologic Drugs Advisory Committee (ODAC) voted 4 in favor and 5 against the risk-benefit profile of UGN-102. This decision marks a major obstacle for UroGen, which has been seeking approval for the drug in the United States. The committee's concerns centered on the drug's safety and efficacy, which did not meet the required standards for approval. This rejection is particularly impactful as UroGen has been actively pursuing regulatory approval to bring this innovative treatment to market.

In October of last year, the FDA accepted UroGen's New Drug Application (NDA) for UGN-102, intended for the treatment of recurrent low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC). The target action date for this application was set for June 13, 2025. Mark Schoenberg, UroGen's Chief Medical Officer, emphasized the high unmet need in this area, stating, "LG-IR-NMIBC is a highly recurrent disease, and many elderly patients often require repeated surgeries under general anesthesia. This is an area of high unmet need, and we believe patients deserve more treatment options."

While the FDA advisory committee's recommendation is not binding, the regulatory body typically considers these opinions before making a final approval decision. This setback for UroGen underscores the stringent standards set by the FDA for approving new cancer treatments, ensuring that only therapies with a proven benefit to patients are brought to market. The company will now need to address the concerns raised by the committee and potentially conduct further clinical trials to demonstrate the safety and efficacy of UGN-102.