The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to UGN-101 (mitomycin gel; UroGen Pharma), as a potential treatment for patients with low-grade upper tract urothelial cancer (LG UTUC).

UGN-101 is an investigational formulation of mitomycin, delivered to patients using standard intravesical catheters. The formulation is hydrogel-based and designed for sustained release to enable longer exposure of mitomycin to the urinary tract tissue, which, the Company says, could “potentially enable the treatment of tumors by non-surgical means.” 

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The treatment was previously granted Orphan and Fast Track designations by the FDA. The Breakthrough Therapy designation is supported by data from the ongoing Phase 3 OLYMPUS clinical trial of UGN-101 for non-surgical treatment of LG UTUC. Interim results showed that 20 of the 34 patients enrolled had a complete response to the treatment; 15% of patients achieved a partial response. Additionally, most treatment-emergent adverse events were characterized as mild or moderate and transient.

“We look forward to close collaboration with the FDA as we bring this potentially transformative therapy to patients with LG UTUC as quickly as possible,” said Mark Schoenberg, MD, CMO of UroGen. 

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