NeoPharm, Inc., announced today that it has filed an orphan drug application with the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) to have Orphan DrugDesignationgranted to IL13-PE38QQR (IL13-PE) for the treatment of Idiopathic Pulmonary Fibrosis (IPF). The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. IPF is the most deadly disease of the lungs in humans with very high morbidity. It is estimated that at least 55,000 patients are diagnosed with IPF each year in America and about 45,000 patients die each year with the disease. There is currently no proven effective treatment for the cure of this lethal disease.
Dr. Aquilur Rahman, President and CEO, commented, “All the studies that NeoPharm has performed in animals and in ex vivo human tissue have shown that IL13-PE works as a targeted therapy for lung fibroblasts. IL13-PE exclusively binds to the pulmonary fibroblasts, which express IL13 receptors for selective cytotoxicity, thereby ameliorating all the clinical and histopathological evidence of IPF. We are looking forward to the start of our clinical studies in humans inflicted with this disease.”
Orphan Drug designation provides a seven-year term of market exclusivity for IPF upon final FDA approval. Orphan Drug designation would position NeoPharm to be able to take advantage of a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees and certain tax credits.