FDA gives mitomycin gel green light for low-grade upper tract urothelial cancer
The approval was based on the Phase III OLYMPUS (NCT02793128) study. The multicenter, single-arm trial enrolled 71 patients with treatment-naïve or recurrent low-grade non-invasive upper tract urothelial cancer (UTUC) with a minimum of one measurable papillary tumor above the ureteropelvic junction.
Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement:
“Due to substantial treatment challenges associated with the complex anatomy of the upper urinary tract, many patients need to be treated with radical surgery – usually complete removal of the affected kidney, ureter and bladder cuff. Jelmyto gives patients, for the first time, an alternative treatment option for low-grade UTUC.”
Mitomycin gel was administered via nephrostomy tube or ureteral catheter once a week for six weeks. Instillations were then administered monthly for patients with a complete response (CR) at three months, for a maximum of 11 additional instillations.
Efficacy was primarily measured in terms of CR and CR durability, with CR being defined as complete absence of tumor lesions three months after mitomycin initiation; this was assessed by urine cytology and ureteroscopy.
41/71 patients (58%) achieved CR three months following the initiation of treatment; 29 received at least one maintenance dose. Following CR determination, response durability was evaluated at three, six, nine, and 12 months. 19 patients remained in CR at 12 months, seven had documented recurrences, and the median response duration was not reached (range: 0, 18.8+ months).
In terms of adverse reactions, the most common were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. Ureteric obstruction occurred in 58% of patients, and 88% of these patients required ureteral stent placement.
Written by Thomas Southgate
U.S Food and Drug Administration. FDA approves mitomycin for low-grade upper tract urothelial cancer (2020). Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mitomycin-low-grade-upper-tract-urothelial-cancer. Last Accessed: 16/04/20.