The FDA’s Oncologic Drugs Advisory Committee narrowly voted against the approval of Zusduri, citing the lack of a completely randomized study to back up the application.
Despite failing to secure the backing of an external panel of advisors, UroGen won the FDA’s approval on Thursday for its intravesical drug mitomycin for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer. UroGen will market the drug under the brand name Zusduri.
Zusduri is the first FDA-approved therapy for this indication and is meant for use in adults with recurrent disease, according to UroGen’s announcement. The drug, which is administered via a catheter directly into the bladder in an out-patient procedure, will be available “on or around” July 1.
UroGen was up nearly 52% when markets closed on Thursday. Zusduri is the company’s second commercial product, following the low-grade upper tract urothelial cancer drug Jelmyto, which was approved in April 2020.
The Phase III ENVISION study, which is still ongoing, supported the regulatory decision with a 79.6% complete response rate at 3 months. The single-arm pivotal trial documented an 80.6% duration of response at 18 months, according to the latest data, released in April.
Despite the promising data, Zusduri’s approval comes as a bit of a surprise, given that the FDA’s Oncologic Drugs Advisory Committee last month recommended against the drug—though only narrowly so. With a 5–4 split, the independent panel of experts pointed to several issues with UroGen’s application package, including the lack of a completely randomized study and the short follow-up in ENVISION.
Perhaps to address these concerns, UroGen has come to a post-marketing commitment agreement with the FDA, the company said in its announcement. UroGen will see the ENVISION trial through to completion “to further characterize the clinical benefit of Zusduri.” The company will also provide the FDA with yearly updates regarding the drug’s duration of response for all treated patients with ongoing complete responses.
On a more sour note, the New York-based Gross Law Firm on Thursday issued a reminder to UroGen shareholders that if they purchased company stock from July 27, 2023 to May 15, 2025 they are eligible to join a class-action lawsuit, which alleges that during that period, UroGen “issued materially false and/or misleading statements” regarding ENVISION.
The lawsuit claims that UroGen did not make it clear to investors that ENVISION “was not designed to demonstrate substantial evidence of effectiveness” of Zusduri and that, as a result, the company “would have difficulty demonstrating that the duration of response endpoint was attributable” to the drug. The company also “failed to heed the FDA’s warning about the study design,” as per the complaint.
The oversight, the lawsuit contends, exposed the company and its shareholder to “substantial risk” that Zusduri would not get approved.
Correction (June 13): This story has been corrected to state that Zusduri is UroGen’s second FDA-approved product. BioSpace regrets the error.
