FDA approves UroGen’s Jelmyto as first therapy for low-grade upper tract urothelial cancer

pharmafile | April 16, 2020 | News story | Sales and Marketing Cancer, FDA, Jelmyto, UroGen Pharma, pharma, urothelial cancer 

The FDA has moved to authorise UroGen Pharma’s Jelmyto (mitomycin gel) in the US for the treatment of low-grade upper tract urothelial cancer (UTUC), marking the first approval of a drug for this specific indication.

UTUC is a cancer of the urinary system, whereas the majority of urothelial cancers occur in the bladder, and can block the kidney or ureter, impairing their function, while tumours associated with the condition are often recurrent. Low-grade UTUC is estimated to affect between 6,000 and 8,000 patients in the US.

The drug secured approval via the Priority Review and Breakthrough Therapy pathway off the back of Phase 3 data which showed that 58% of 71 patients treated with the drug demonstrated a complete response three months after initial treatment.

Patients were also assessed every three months for a year via urine cytology, ureteroscopy and biopsy to determine durability of response, and it was found that 46% of patients who achieved a complete response maintained it for 12 months.

“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,” commented Dr Richard Pazdur, 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. “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.”

The FDA ruling did note however that use of Jelmyto can cause serious side-effects such as urosepsis, flank pain, and ureteric obstruction. In the case of the latter, patients may need transient or long-term treatment with stents; in the Jelmyto trial, 51% of patients who experienced such obstruction did not see the condition resolve completely.

Dr Pazdur added: “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. 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.”

Matt Fellows

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