FDA approves Jelmyto for low-grade upper tract urothelial cancer
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The FDA approved mitomycin gel for the treatment of low-grade upper tract urothelial cancer.
Mitomycin gel (Jelmyto, UroGen Pharma) is an alkylating drug that inhibits the transcription of DNA into RNA, stopping protein synthesis and prohibiting the cancer cell from multiplying.
“This is the first approval specifically for patients with low-grade [upper tract urothelial cancer] and provides an option for some patients who may otherwise require a nephroureterectomy,” 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, said in a press release. “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 [upper tract urothelial cancer].”
As Healio previously reported, the FDA previously granted priority review designation to mitomycin gel for treatment of low-grade upper tract urothelial cancer. The agency also granted breakthrough therapy, orphan drug and fast track status to the agent for the same indication.
The FDA based its approval on results of the phase 3 OLYMPUS trial, which evaluated 71 patients with low-grade upper tract urothelial cancer who had not been previously treated for their disease. Complete response at 3 months after initiation of therapy served as the study’s primary endpoint.
Results showed 58% of patients (n = 41) achieved complete response after six weekly treatments of mitomycin gel. Nineteen of these patients (46%) continued to demonstrate complete response at 12 months.
The most frequently reported treatment-emergent adverse events, as previously reported by Healio, included ureteric stenosis (43.7%), urinary tract infection (32.4%), hematuria (31%), flank pain (29.6%), nausea (23.9%), dysuria (21.1%), renal impairment (19.7%) and vomiting (19.7%).
The FDA recommended women who are pregnant not use mitomycin gel because it may harm a developing fetus or newborn baby. The agency also advised health care professionals to recommend use of effective contraception by men and women during treatment with mitomycin gel.
“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,” Pazdur said. “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.”