FDA Granted Orphan Drug Designation to Novel ImPACT Agent in Upper Tract Urothelial Cancer

The FDA has granted an Orphan Drug Designation to Padeliporfin Immune Photo Activated Cancer Therapy (ImPACT) for the treatment of adult patients with upper tract urothelial cancer, according to a press release from Steba Biotech.

“We are delighted to receive Orphan Drug Designation for Padeliporfin ImPACT in upper tract urothelial cancer, further validating Steba’s technology and the benefits our product could bring to people suffering from upper tract urothelial cancer. It has been less than a year since we embarked on our new strategy, refocusing Padeliporfin ImPACT on diseases for patients with limited treatment options,” said Barack Palatchi, chief executive officer, Steba Biotech, in a statement.

Following an Investigation New Drug Application, which was accepted by the FDA, a phase 3 clinical trial (ENLIGHTED; NCT04620239) has been initiated to further study the use of Padeliporfin ImPACT. Enrollment is expected to begin later this month.

ENLIGHTED is a single-arm, non-randomized, multicenter study with a target enrollment of 100 patients with low-grade upper tract urothelial cancer. The primary end point of the study is the number of patients with the absence of upper tract urothelial cancer tumors in the entire ipsilateral renal pelvis and ureter. The secondary end points of the study include the duration of response at the entire ipsilateral kidney, at the entire ipsilateral kidney, and at the treatment area of the ipsilateral kidney; overall renal function; the number of patients with kidney organ loss or preservation; and the number of patients with ureteral obstruction.

Padeliporfin ImPACT is an endoluminal light-activated treatment that involves the placement of optical light fiber with a ureteroscope and intravenous administration of Padeliporfin at 4 mg/kg over 10 minutes and the illumination of each area for 10 minutes in the study. The treatment triggers the constriction of the blood supply in the affected area upon activation and leads to targeted tumor necrosis and antitumor immunity.

Those participating in the study will receive 1 to 3 treatments with Padeliporfin ImPACT given 4 weeks apart as induction in order to try to achieve complete response in the involved ipsilateral tract system. Patients with evidence of treatable tumor recurrence will be allowed to receive repeated maintenance treatments.

Patients eligible to receive Padeliporfin ImPACT in the ENLIGHTED study are those aged 18 years of age or older with new or recurrent low-grade, non-invasive upper tract urothelial cancer that has been proven by biopsy in up to 2 sites. Tumors in patients enrolled must be between 5 and 15 mm in diameter. All patients are also required to have a Karnofsky performance status ≥ 50% and adequate organ function.

Individuals who have a life expectancy of fewer than 2 years, current high-grade or muscle-invasive urothelial cancer of the bladder, current or previous carcinoma in situ in the upper urinary tract, a history of invasive T2 or higher urothelial cancer within 2 years of the study, and those with any medical or psychiatric comorbidities are not eligible for ENLIGHTED. In terms of prior treatment, patients who were participants in another clinical study involving an investigational product within 1 month before study entry and those who had systemic chemotherapy treatment within 2 months prior to study entry were excluded from the study.

“In upper tract urothelial cancer, the current standard of care is nephrectomy; our hope is that Padeliporfin ImPACT offers these patients an effective treatment option that preserves their kidney. Upper tract urothelial cancer is the first indication of our ambitious 3-year plan. We are looking forward to exploring the possibility of offering Padeliporfin ImPACT to more patients coping with life-threatening cancers,” Palatchi added.

The treatment also previously received Fast Track designation from the FDA for the treatment of adult patients with low-grade and unifocal high-grade upper tract urothelial cancer in January 2021.

_Reference:

_{Steba Biotech receives FDA Orphan Drug Designation for Padeliporfin ImPACT in upper tract urothelial cancer. News release. Steba Biotech. March 8, 2021. Accessed March 8, 2021. https://bit.ly/3qsWfDA}