FDA Approves UroGen's Bladder Cancer Treatment After ODAC Rebuff

The FDA has granted approval to UroGen Pharma's Zusduri for treating recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). This decision comes shortly after the Oncologic Drugs Advisory Committee (ODAC) narrowly voted against the drug's benefit-risk profile. The committee's 5-4 vote raised concerns, particularly due to the absence of a control arm in the pivotal trial and skepticism regarding the durability of the drug's effects. Zusduri, which was previously known as UGN-102, is based on mitomycin and is delivered intravesically with the aid of UroGen's RTGel technology. This formulation allows for prolonged local drug delivery directly into the bladder in an outpatient setting.

The FDA's decision was supported by the results from the Phase III ENVISION study, where Zusduri achieved a 78% complete response rate at the three-month mark. Among those who responded, 79% maintained a complete response one year later. The safety profile was deemed manageable, with primary concerns involving lower urinary tract symptoms.

"For the first time, approximately 59,000 patients in the US dealing with recurrent LG-IR-NMIBC each year have access to an FDA-approved medication," noted CEO Liz Barrett. The announcement led to a significant surge in UroGen's stock, which rose by more than 51% following the news.

The approval of Zusduri could herald a shift away from the conventional surgical method known as transurethral resection of bladder tumor (TURBT). This procedure is often repeated in patients due to the high recurrence rates of the disease. Analysts from D. Boral Capital remarked that "Zusduri offers a significant therapeutic alternative in a field with few non-surgical options, and it validates UroGen's broader intravesical platform."

As part of its post-marketing obligations, UroGen has committed to completing the ongoing ENVISION trial to provide more comprehensive insights into the clinical benefits of Zusduri. Additionally, the company has pledged to deliver annual updates concerning the durability of the drug’s response. UroGen has announced that Zusduri will be available in the US market starting around July 1.

This approval is particularly noteworthy given the initial skepticism following the ODAC meeting. Nevertheless, it underscores the potential for innovation in a space that has seen limited advancement in non-surgical treatments. The introduction of Zusduri could potentially reduce the reliance on surgical procedures for patients with recurrent LG-IR-NMIBC, offering them a more convenient and less invasive treatment option. As UroGen fulfills its commitment to ongoing research and monitoring, future updates will likely provide further insights into the long-term efficacy and safety of this new treatment option.