FDA Approves Mitomycin Gel for UTUC

The FDA approved mitomycin gel (Jelmyto) for the treatment of patients with low-grade upper tract urothelial cancer (UTUC), cancer of the lining of the urinary system. This is the first therapy approved in this space.

“Although our nation’s emphasis is on the need to combat COVID-19, patients with cancer and their unique needs continue to be a top priority for the FDA,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a statement.

“We continue to expedite oncology product development in this critical time. Our staff is continuing to meet virtually with drug developers, academic investigators and patient advocates to push forward the coordinated review of drugs, biologics and devices for cancer.”

The approval was based on the results of a clinical trial that involved 71 patients with low-grade UTUC who had undergone treatment or had recurrent, low-grade non-invasive UTUC with 1 or more papillary tumor. Participants received mitomycin once a week for 6 weeks and if they were assessed as a complete response, they had the drug monthly for up to an additional 11 months. The effectiveness of the drug was evaluated using urine cytology, ureteroscopy, and biopsy (if warranted) 3 months after the initiation of treatment.

The study’s primary endpoint was complete response at 3 months after initiation of therapy. Fifty-eight percent (n= 41) had a complete response after 6 treatments. Durability was also examined every 3 months for a year after the start of treatment. Forty-six percent (n= 19) who had a complete response continued to have a complete response at the 12-month mark.

Common adverse events (AEs) for mitomycin included flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria and vomiting. More serious AEs were ureteric obstruction, flank pain and urosepsis (bacteria in the bloodstream from a urinary tract infection).

Nurses should monitor for ureteric obstructions, as they require transient or long-term stents to relieve this obstruction. The obstruction may be persistent and did not resolve or did not resolve completely in 51% of patients who experienced obstruction due to mitomycin. The drug should be avoided in patients with a glomerular filtration rate of less than 30mL/min or in pregnant women.

“This is the first approval specifically for patients with low-grade UTUC and provides an option for some patients who may otherwise require a nephroureterectomy,” said Pazdur. “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.”