HedgePath Pharmaceuticals, Inc., a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative therapeutics for patients with cancer, announced that it has received notice of Orphan Drug Designation for treatment of patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS) with its oral formulation of SUBA-Itraconazole Capsules.
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.
"We believe BCCNS provides us with an ideal first opportunity to test SUBA-Itraconazole's anti-cancer potential," said Nicholas J. Virca, President & CEO of HedgePath Pharmaceuticals. "There is a relatively well-defined molecular mechanism associated with BCCNS that causes patients to suffer from multiple, chronically recurring tumors which can be debilitating and often require disfiguring surgeries to remove cancerous lesions. Existing topical therapies have proven suboptimal due to lack of efficacy, and marketed oral therapies for BCC are only approved for locally advanced or metastatic basal cell carcinomas that are not candidates for radiation or surgery and carry significant side-effects that limit their chronic use. We believe that our Phase II (b) trial design lays out a well-defined regulatory pathway, as we continue to move forward developing what we believe will be an effective treatment for this underserved patient population."
HPPI anticipates that, based upon evidence of substantial clinical benefit in subjects currently enrolled in our clinical trial, the company will seek to have further discussions with FDA as to whether results from this trial could potentially serve as the basis for a New Drug Application submission to FDA.