Low-grade upper tract urothelial cancer (LG UTUC) is a malignancy involving the lining of the ureters, renal pelvis and kidneys. It is a rare but critical disease affecting approximately 3,000 new patients in the United States each year. Approximately 6,000 to 8,000 patients present with new or recurrent LG UTUC every year.
There are currently no drugs approved by the FDA for the treatment of LG UTUC, representing a significant unmet medical need. The anatomical complexity of the upper urinary tract, particularly the renal pelvis, presents significant challenges, and as a result, patients with high-grade disease or patients with low-grade disease that present with large tumors or multifocal disease typically undergo radical nephroureterectomy (RNU), which is removal of the kidney and upper urinary tract. Endoscopic tumor resection, which aims to be a kidney sparing surgical procedure, is conducted only in patients with low-grade disease that present with a limited number of tumors that can be easily visualized and destroyed using standard instrumentation. However, the upper urinary tract’s anatomical constraints limit the effectiveness of surgical procedures and adjuvant chemotherapy treatments, leading to high rates of recurrence in this patient population.
UGN-101 (mitomycin gel) for instillation, 0.4%, is an investigational new drug formulation that utilizes the RTGel™ technology platform. The Company recently completed enrollment for a Phase 3 trial to assess the safety and efficacy of UGN-101 for the treatment of patients with LG UTUC. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of LG UTUC. In December 2018, the Company initiated a rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and intends to complete the submission in 2019. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.
*Margulis 2009; Cutress 2010; Primary Market Research
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