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News From the Food and Drug Administration
May 19, 2020

Therapy Approved for Hard-to-Treat Upper Tract Urothelial Cancer

JAMA. 2020;323(19):1887. doi:10.1001/jama.2020.7153

The first treatment for low-grade upper tract urothelial cancer (UTUC) has received FDA approval.

Although US health officials were focused on combating coronavirus disease 2019 when mitomycin gel was approved in mid-April, the director of the FDA’s Oncology Center of Excellence said that “patients with cancer and their unique needs continue to be a top priority” for the agency.

Richard Pazdur, MD, said in a statement that the agency continued to expedite oncology product development during the pandemic. “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,” he noted.

Mitomycin gel, marketed as Jelmyto, is an alkylating drug—it inhibits the transcription of DNA into RNA, which stops protein synthesis and eliminates a cancer cell’s ability to multiply. Most urothelial cancers occur in the bladder, but UTUC represents a small subset that arise in the lining of the kidney or the ureter. If UTUC blocks the ureter or kidney, swelling, infection, and impaired kidney function may result.

The condition is rare, affecting about 6000 to 7000 patients annually in the US, according to a statement from the drug’s manufacturer, UroGen Pharma Ltd of Princeton, New Jersey. But it is also challenging to treat because of the urinary tract system’s complex anatomy. Patients often require multiple surgeries, including radical nephroureterectomy—removal of the renal pelvis, kidney, ureter, and bladder cuff.

Approval of mitomycin gel, which is administered via standard ureteral catheters, was based on a single-group phase 3 clinical trial involving 74 treatment-naive patients at clinical sites in the US and Israel, according to the UroGen statement. Patients received a weekly infusion for 6 weeks. Those who achieved a complete response 4 to 6 weeks after the last infusion were followed up for 12 months. Among the 58% of patients who were followed up, 46% maintained a complete response at 12 months.

Common adverse events included ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting.