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FDA grants orphan drug status to RX-3117 for pancreatic cancer
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Rexahn Pharmaceuticals announced that the FDA granted orphan drug designation to its cancer-specific nucleoside analog, RX-3117, for the treatment of patients with pancreatic cancer.
In preclinical research, RX-3117 demonstrated an ability to overcome cancer drug resistance in cancer cells, most notably gemcitabine-resistance in human lung cancer cells. Other potential indications of RX-3117 include solid tumors, such as colon, bladder and pancreatic cancers.
The FDA based its decision in part on preliminary research from a phase 1b multi-center dose-escalation study intended to evaluate the safety, tolerability, dose-limiting toxicities and maximal tolerated dose of RX-3117 in patients with solid tumors.
Secondary endpoints from this study will include illustrating the pharmacokinetic profile of RX-3117 and assessing its initial anti-tumor effects.
“Receiving Orphan Drug Designation for RX-3117 in the treatment of pancreatic cancer is an important milestone for this clinical development program,” Peter D. Suzdak, PhD, CEO of Rexahn Pharmaceuticals, said in a press release. “RX-3117 has shown to be effective in treating cancer cells that have become gemcitabine resistant. If the results of our phase 1b trial and future trials show similar results in gemcitabine resistant patients, this would represent a major breakthrough in the treatment of pancreatic cancer patients.”
The FDA Office of Orphan Products grants orphan drug designation to novel drugs and biologics 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 United States.