30 October 2018 - On track to initiate rolling submission of UGN-101 new drug application in Q4 2018.

UroGen Pharma today announced that the U.S. FDA has granted breakthrough therapy designation status to the Company’s lead product candidate, UGN-101, (mitomycin gel) for instillation. UGN-101 is currently in Phase 3 development for the treatment of patients with low-grade upper tract urothelial cancer (LG UTUC). The FDA previously granted both orphan drug and fast track designations to UGN-101 for the treatment of LG UTUC.

The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates that use of the drug may result in substantial improvement on at least one clinically significant endpoint over available therapy. The Breakthrough Therapy Designation for UGN-101 is supported by data from the ongoing Phase 3 OLYMPUS clinical trial of UGN-101 for the non-surgical treatment of LG UTUC. Results from an interim analysis presented in May, 2018 showed a complete response (CR) rate of 59 percent (20 out of the interim analysis intent to treat population of 34 patients) who were evaluated for primary disease evaluation (PDE, or the primary endpoint). In addition, 15 percent (five of 34 patients) achieved a partial response. At the time of the interim analysis presentation, of the 20 patients who achieved a CR, 13 patients had reached three-month follow-up, and all remained in CR. Four of these 13 patients had reached six-month follow-up and one of the 13 patients had reached nine-month follow-up, and all remained in CR. UGN-101 appeared to be well-tolerated with most treatment-emergent adverse events characterized as mild or moderate and transient.

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