FDA grants Orphan Drug designation to padeliporfin ImPACT for UTUC

The investigational padeliporfin ImPACT platform involves the intravenous delivery of the photosensitive drug padeliporfin, which is then activated by a non-thermal light delivery system.

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 (UTUC), according to Steba biotech, the developer of the investigational treatment.1

The padeliporfin ImPACT platform involves the intravenous delivery and activation of the photosensitive drug padeliporfin. Once the drug is in the patient, it is “activated by a non-thermal light delivery system that uses a lower energy laser light channeled through optical fibers.”2 The minimally invasive approach enables targeted ablation of the tumor. The treatment was initially explored in prostate cancer and is now being investigated in UTUC and other solid tumors.

Padeliporfin ImPACT has shown positive initial efficacy in prostate cancer and a phase 1 UTUC trial. In December 2020, Steba received FDA clearance on an Investigational New Drug application, permitting the launch of a phase 3 study of padeliporfin ImPACT in patients with low-grade UTUC. The study is expected to start enrollment by the end of this month.

The FDA previously granted Fast Track designation to padeliporfin ImPACT for the treatment of adult patients with low-grade and unifocal high-grade UTUC. The Fast Track and Orphan Drug designations are designed to expedite the review and development of novel treatments that will fill an unmet medical need.

“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. In UTUC, the current standard of care is nephrectomy; our hope is that padeliporfin ImPACT offers these patients an effective treatment option that preserves their kidney. UTUC 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,” Barak Palatchi, CEO of Steba Biotech, stated in a press release.

A major breakthrough in the UTUC armamentarium came in April 2020 when the FDA approved Jelmyto (mitomycin gel) as the first therapy to treat low-grade UTUC.3

According to the FDA, UTUC includes a small subset of urothelial tumors that occur in the lining of the kidney or the ureter. The low-grade subtype of UTUC is most commonly noninvasive and confined to the kidney or ureter; however, these tumors frequently recur.

Overall about 6000 to 8000 new cases of low-grade UTUC occur annually in the United States. Despite the major breakthrough with Jelmyto, there remains a high unmet need for novel UTUC treatments.


1. Steba Biotech receives FDA Orphan Drug Designation for Padeliporfin ImPACT in Upper Tract Urothelial Cancer. Posted online March 8, 2021. Accessed March 8, 2021. https://bit.ly/3rtDXDv.

2. Steba biotech. A Novel Treatment Path. Accessed March 8, 2021. https://www.stebabiotech.com/technology-innovation.

3. FDA Approves First Therapy for Treatment of Low-Grade Upper Tract Urothelial Cancer. Posted online April 15, 2020. Accessed January 19, 2021. https://bit.ly/2XUxWCD.