Investing.com -- UroGen Pharma (NASDAQ: URGN ) shares plummeted 29% following concerns from FDA staff regarding the efficacy of its bladder cancer drug, UGN-102. The FDA’s briefing papers, released ahead of the advisory panel review set for May 21, cast doubt on the experimental therapy for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

The FDA staff challenged the company’s claims about UGN-102’s efficacy, specifically disputing that the complete response rate for the drug was higher or more durable compared to the current standard of care, Transurethral resection of bladder tumor (TURBT). Additionally, the FDA questioned the long-term benefits of UGN-102, citing a lack of data on future treatments and its potential to reduce the frequency of TURBT procedures in certain patient populations.

Oppenheimer analyst Leland Gershell maintained an Outperform rating and a $36.00 price target on UroGen Pharma, but acknowledged that the FDA documents were a cause for concern. Gershell highlighted the FDA’s issues with the single-arm pivotal trial design and the inability to adequately assess UGN-102’s efficacy. "They read more negatively vs our expectations, with the FDA expressing particular concern around the single-arm pivotal trial design and its limited ability to sufficiently inform on UGN-102’s efficacy, as viewed independently and in relation to the standard of care (TURBT)," Gershell stated.

Despite the negative briefing documents, TD Cowen analyst Tara Bancroft expressed confidence in a positive advisory panel vote and subsequent drug approval, emphasizing the company’s preparedness for the panel discussions.

Investors reacted sharply to the FDA’s briefing, leading to the significant drop in UroGen’s stock price. The market’s response underscores the critical nature of FDA evaluations in determining the commercial viability of new pharmaceuticals and the high stakes for companies navigating the approval process. The outcome of the May 21 advisory panel meeting will be closely watched, as it will likely have a substantial impact on UroGen’s future prospects.

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