FDA Gives UroGen’s Jelmyto Expedited Approval for Urothelial Cancer


The U.S. Food and Drug Administration (FDA) approved UroGen Pharma’s Jelmyto (mitomycin) for pyelocaliceal solution for adults with low-grade upper tract urothelial cancer (LG UTUC). It was granted expedited approval on the basis of data from the Phase III OLYMPUS trial.

Jelmyto is made up of mitomycin, an established chemotherapy agent, and sterile hydrogel, using the company’s proprietary sustained release RTGel technology. It is designed to allow longer exposure of urinary tract tissue to mitomycin. This is the first and only non-surgical treatment for patients with this indication.

“UroGen was founded on the vision to improve lives by challenging the current standard of care,” said Liz Barrett, president and chief executive officer of UroGen. “Jelmyto, which leverages our innovative technology and expertise in specialty cancers and urologic diseases, is just the beginning as we build a company focused on bringing novel solutions to patients.”

Urothelial cancer is the most common form of bladder cancer. UTUC is a cancer of the lining of the urinary tract, meaning the lining of the kidney or the ureter, the tube that runs between the kidney and the bladder. About 6,000 to 7,000 patients are diagnosed with low-grade UTUC annually, with care mostly treating visible tumors and working to save the urinary tract. This typically means multiple surgeries with most patients requiring radical nephroureterectomy, meaning removal of the renal pelvis, kidney, ureter and bladder cuff. It is most commonly diagnosed in patients over 70 years of age.

The Phase III OLYMPUS trial demonstrated the drug achieved clinically significant eradication in adults with LG UTUC. It evaluated 71 patients. There was a complete response (CR) in 58% of the intent-to-treat population and in a sub-group of patients who were ineligible for surgical removal at diagnosis. At the one-year mark, 19 patients remained in CR, seven had recurrence, and nine were followed for the 12-month duration of response.

“There has been little progress for decades in the treatment of low-grade upper tract urothelial cancer, and this new option is paradigm-shifting for patients who often face recurrence and major surgery to remove their kidney,” said Andrea Maddox-Smith, chief executive officer of the Bladder Cancer Advocacy Network. “We applaud the FDA approval and the impact it will have for this community of elderly patients, many of whom struggle with comorbidities and have been hoping for a surgery-free treatment option.”

The company is also working to advance the drug for low-grade intermediate risk non-muscle invasive bladder cancer. Another of its drugs is being studied by Allergan in a Phase II trial in combination with Botox for overactive bladder. UroGen’s pipeline includes an immunotherapy, UGN-201, for high-grade non-muscle invasive bladder cancer (NMIBC).

Of the approval, Seth Lerner, professor of Urology at Baylor College of Medicine in Houston, and principal investigator of the OLYMPUS trial, said, “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. 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.”


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