FDA News

FDA grants breakthrough therapy designation to UGN-101 for urothelial cancer

The FDA granted breakthrough therapy designation to UGN-101 for the treatment of patients with low-grade upper tract urothelial cancer, according to the agent’s manufacturer.

UGN-101 (UroGen Pharma) — an investigational drug formulation of mitomycin — is in phase 3 development for this indication.

UGN-101 is delivered via standard intravesical catheters.

The sustained release, hydrogel-based formulation is intended to allow for longer exposure of mitomycin to the urinary tract tissue, enabling treatment of tumors by means other than surgery.

“We are very excited about receiving the breakthrough therapy designation for UGN-101 and the potential to deliver this far less invasive, organ-sparing therapy option to patients,” Ron Bentsur, CEO of UroGen, said in a company-issued press release. “We look forward to working with the FDA as we prepare to initiate a rolling submission of the UGN-101 new drug application later this year, with the potential to become the first drug ever approved as frontline treatment of low-grade upper tract urothelial cancer.”

Low-grade upper tract urothelial cancer is a rare malignant tumor of the cells that line the urinary tract. It is most common among elderly individuals who have comorbid conditions, such as diabetes, obesity, hypertension and metabolic syndrome.

Approximately 6,000 to 8,000 people in the United States develop new or recurrent low-grade upper tract urothelial cancer every year.

Standard treatment typically consists of complete removal of the kidney and/or partial removal of the ureter. Repetitive endoscopic tumor resection is used when feasible for patients with limited tumor burden.

These surgical interventions carry risks for infection and bleeding, as well as injury to adjacent organs and potential long-term morbidity associated with removal of the kidney.

“UGN-101 was developed to provide an effective alternative to current treatment options, that avoids the risks of surgery, anesthesia and the deleterious effects of kidney removal,” Mark Schoenberg, MD, UroGen’s chief medical officer, said in the release. “The breakthrough therapy designation confirms that UGN-101 represents a novel and effective approach to treat this devastating disease, and we look forward to close collaboration with the FDA as we bring this potentially transformative therapy to patients ... as quickly as possible.”

The FDA granted breakthrough therapy designation to UGN-101 for the treatment of patients with low-grade upper tract urothelial cancer, according to the agent’s manufacturer.

UGN-101 (UroGen Pharma) — an investigational drug formulation of mitomycin — is in phase 3 development for this indication.

UGN-101 is delivered via standard intravesical catheters.

The sustained release, hydrogel-based formulation is intended to allow for longer exposure of mitomycin to the urinary tract tissue, enabling treatment of tumors by means other than surgery.

“We are very excited about receiving the breakthrough therapy designation for UGN-101 and the potential to deliver this far less invasive, organ-sparing therapy option to patients,” Ron Bentsur, CEO of UroGen, said in a company-issued press release. “We look forward to working with the FDA as we prepare to initiate a rolling submission of the UGN-101 new drug application later this year, with the potential to become the first drug ever approved as frontline treatment of low-grade upper tract urothelial cancer.”

Low-grade upper tract urothelial cancer is a rare malignant tumor of the cells that line the urinary tract. It is most common among elderly individuals who have comorbid conditions, such as diabetes, obesity, hypertension and metabolic syndrome.

Approximately 6,000 to 8,000 people in the United States develop new or recurrent low-grade upper tract urothelial cancer every year.

Standard treatment typically consists of complete removal of the kidney and/or partial removal of the ureter. Repetitive endoscopic tumor resection is used when feasible for patients with limited tumor burden.

These surgical interventions carry risks for infection and bleeding, as well as injury to adjacent organs and potential long-term morbidity associated with removal of the kidney.

“UGN-101 was developed to provide an effective alternative to current treatment options, that avoids the risks of surgery, anesthesia and the deleterious effects of kidney removal,” Mark Schoenberg, MD, UroGen’s chief medical officer, said in the release. “The breakthrough therapy designation confirms that UGN-101 represents a novel and effective approach to treat this devastating disease, and we look forward to close collaboration with the FDA as we bring this potentially transformative therapy to patients ... as quickly as possible.”