Urogen announced the U.S. FDA has accepted for filing and granted priority review for its New Drug Application (NDA) for UGN-101 (mitomycin gel) for instillation as a potential treatment for patients with low-grade upper tract urothelial cancer (LG UTUC). The FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of April 18, 2020. If approved, UGN-101 would be the first non-surgical treatment option for LG UTUC.
The FDA grants priority review to applications for medicines that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Priority review designation shortens the review period from the standard 10 months to six months from the submission of the NDA.
The NDA is supported by the positive results from the pivotal Phase 3 OLYMPUS clinical trial. OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is a pivotal, open-label, single-arm trial of UGN-101 (mitomycin gel) for instillation to evaluate the safety, tolerability and tumor ablative effect of UGN-101 in patients with low-grade UTUC. The trial enrolled 74 patients at clinical sites across the United States and Israel. The patients were treated with six weekly instillations of UGN-101 administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine complete response (CR), the primary endpoint of the study.
Results from a final analysis of the primary endpoint showed that UGN-101 demonstrated a complete response rate of 59 percent in patients with LG UTUC. In addition, the durability of response was estimated as 89 percent at six months and 84 percent at 12 months by Kaplan Meier analysis. Median time to recurrence was estimated to be 13 months. The most common adverse events were ureteric stenosis, urinary tract infection, hematuria, flank pain, nausea, dysuria, renal impairment and vomiting.
The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of LG UTUC.
UGN-101 (mitomycin gel) for instillation is a drug formulation of mitomycin for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel™ technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters.
About Low-grade UTUC
Low-grade UTUC is a urothelial malignancy that presents in the lining of the upper urinary tract, in the ureters and renal collecting system of the kidneys. It is a rare but critical cancer that affects approximately 6,000-8,000 new patients in the United States every year.