Padeliporfin ImPACT Receives Orphan Drug Designation for Upper Tract Urothelial Cancer

Upper tract urothelial cancer (UTUC) occurs in the lining of the kidney known as the renal pelvis or the ureter, which can develop as low- or high-grade tumors.Low-grade tumors are not invasive, but they frequently recur and can spread.However, high-grade tumors can spread and are usually treated with nephroureterectomy (surgical removal of the kidney and ureter).Steba Biotech’s novel investigational therapy Padeliporfin ImPACT (Immune Photo Active Cancer Treatment) was recently granted Orphan Drug Designation by the FDA for the treatment of adult patients with UTUC, which provides a variety of incentives — research grants, tax credits, protocol assistance — for the development of drugs for rare diseases.Previously, Padeliporfin ImPACT also received Fast Track designation for the treatment of low-grade and unifocal high-grade UTUC.

In April 2020, mitomycin gel (Jelmyto) became the first FDA approved therapy to treat low-grade UTUC.With limited treatment options available for low-grade UTUC, which is rare but still affects 6,000-8,000 new patients in the U.S. annually, oncologists see an unmet need for nonsurgical therapies for the condition.

ImPACT works by providing intravenous (IV) delivery of the photosensitive drug padeliporfin, which becomes activated as a targeted nonsurgical treatment option.

In a press release, Barak Palatchi, CEO of Steba Biotech said “Our hope is that Padeliporfin ImPACT offers these patients an effective treatment option that preserves their kidney.”This innovative treatment option could help to preserve the kidneys in patients with UTUC where surgery is not the desired course.The FDA has given the go ahead for the ENLIGHTED phase 3 open-label multicenter trial in adult patients with UTUC.

Patients will be treated with padeliporfin in induction and maintenance treatment phases, with an expected enrollment of 100 study participants.Patients will receive IV administration of padeliporfin 3.66 mg/kg infused over 10 minutes, and each target area will be illuminated for 10 minutes.The induction phase involves 1-3 treatments given 4 weeks apart.Patients who achieve a complete response in the induction phase will move onto the maintenance treatment phase and be followed for 12 months.The primary outcome is the number of patients without UTUC tumors approximately 28 days after the last treatment.