FDA Approves First Non-Surgical Treatment for Rare Urinary Tract Cancer

Rare Daily Staff

U.S. Food and Drug Administration granted expedited approval for UroGen Pharma’s Jelmyto, a first-in-class treatment indicated for adults with low-grade upper tract urothelial cancer.

The approval is based on positive results from the phase 3 OLYMPUS trial that showed Jelmyto provides an effective, kidney-sparing option for patients with this rare and difficult-to-treat cancer. The trial met the primary endpoint of complete response of 58 percent in the intent-to-treat population and in the sub-population of patients who were deemed not capable of surgical removal at diagnosis.

Jelmyto consists of the chemotherapy mitomycin and sterile hydrogel, using UroGen’s proprietary sustained release technology, and is inserted into the site of the cancer through a catheter. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.

Low-grade upper urothelial cancer (LG UTUC) is a rare cancer that develops in the lining of the upper urinary tract, ureters, and kidneys. There are approximately 6,000 to 7,000 new or recurrent LG UTUC patients annually in the United States. It is a challenging condition to treat due to the complex anatomy of the urinary tract system. The current standard of care includes multiple surgeries and most patients require a radical nephroureterectomy, which includes the removal of the renal pelvis, kidney, ureter and bladder cuff. Treatment is further complicated by the fact that LG UTUC is most commonly diagnosed in patients over 70 years of age, who may already have compromised kidney functionality and may suffer further complications as a result of major surgery.

“Jelmyto offers a new, non-surgical treatment approach for patients who otherwise may require treatment by radical nephroureterectomy, which is associated with declining kidney function and other complications,” said Seth Lerner, professor of urology at Baylor College of Medicine in Houston, Texas and principal investigator of the OLYMPUS trial. “This novel, minimally invasive, kidney-sparing treatment has the potential to transform the way low-grade upper tract urothelial cancer is treated and help patients avoid long-term complications associated with surgery and the loss of their kidney.”

The FDA had granted Jelmyto Breakthrough Therapy designation, which expedites the development and review of drugs developed for serious or life-threatening conditions with high unmet need, and was evaluated under Priority Review, which is reserved for medicines that may represent significant improvements in safety or efficacy in treating serious conditions.