FDA approves Jelmyto to treat low-grade upper tract urothelial cancer Urogen Pharma

UroGen Pharma Ltd.has announced the FDA granted expedited approval for Jelmyto (mitomycin) for pyelocalyceal solution, a first-in-class treatment indicated for adults with low-grade upper tract urothelial cancer (LG UTUC). This landmark approval is based on positive results from the Phase III OLYMPUS trial that showed Jelmyto provides an effective, kidney-sparing option for patients with this rare and difficult-to-treat cancer. Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using UroGen’s proprietary sustained release RTGel technology. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.

The FDA approval is based on results from the Phase III OLYMPUS trial showing Jelmyto achieved clinically significant disease eradication in adults with LG UTUC. Findings include: Complete response (CR) (primary endpoint) of 58% in the intent-to-treat population and in the sub-population of patients who were deemed not capable of surgical removal at diagnosis. At the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease and nine patients continued to be followed for the 12-month duration of response.

Kaplan-Meier analysis estimated 12-month durability at 84% (based on interim data).The most commonly reported adverse events ( greater than 20%) were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. Most adverse events were mild to moderate and manageable using well established treatments. No treatment-related deaths occurred.