The FDA has granted priority review to a New Drug Application (NDA) for a mitomycin gel formulation to treat low-grade upper tract urothelial cancer (UTUC). If approved, the product would be the first non-surgical treatment for malignancy, according to a press release from UroGen Pharma Ltd, which is developing the gel.
Priority review designation shortens the review period from the standard 10 months to 6 months from the submission of the application, the press release explained. The FDA grants priority review to applications for drugs 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.”
According to UroGen, the company is on track for a potential launch of the product by mid-2020. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to the mitomycin gel for treating low-grade UTUC.
The NDA is supported by the positive results from the pivotal Phase 3 OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) trial, a pivotal, open-label, single-arm phase 3 clinical trial of the mitomycin gel. The trial enrolled 74 patients with low-grade UTUC at clinical sites across the United States and Israel. Study participants received 6 weekly instillations of the gel administered via a standard catheter. Four to 6 weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine complete response (CR), the primary endpoint of the study.
The mitomycin gel demonstrated a CR rate of 59% of patients. The durability of response was estimated to be 89% and 84% at 6 and 12 months, respectively. The estimated median time to recurrence was 13 months.
The most commonly reported treatment-related emergent adverse events (AEs) were ureteric stenosis (43.7%), urinary tract infection (32.4%), haematuria (31.0%), flank pain (29.6%) nausea (23.9%), dysuria (21.1%), renal impairment (19.7%) and vomiting (19.7%), the press release noted. Most AEs were mild to moderate.
UroGen Pharma press release. UroGen Pharma Announces FDA Filing Acceptance and Priority Review of U.S. New Drug Application (NDA) for UGN-101.