InterMune, Inc announced that the Company's investigational drug pirfenidone has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of idiopathic pulmonary fibrosis (IPF), a disease characterized by progressive scarring or fibrosis of the lungs, which ultimately results in death.
"This is an important milestone for InterMune as we continue to advance our pirfenidone development program," said Dan Welch, InterMune's CEO and president. "In 2004, we expect to complete the pre-clinical and manufacturing activities and the data analysis required to initiate our IPF registration program. With the recent launch of the Actimmune Phase III, INSPIRE Trial, we believe that we are developing the two most promising and advanced compounds for the treatment of patients with IPF."
Orphan drug designation is granted to drug candidates targeting rare diseases or conditions. This designation provides seven years market exclusivity in the United States upon FDA's first approval of the product for the orphan indication provided that the sponsor complies with certain FDA- specified conditions. Other additional incentives include tax credits for amounts spent on human clinical trials and exemptions from user fee payments.
"There is promising clinical evidence to suggest that pirfenidone may offer benefit to IPF patients," said Ganesh Raghu, M.D., Professor of Medicine, University of Washington Medical Center, Seattle, WA. "Orphan drug designation is a significant step on the path to further clinical development. I look forward to working closely with InterMune, a committed partner and an innovator in the IPF field, to continue to address this seriously underserved disease."
"Due to the complex nature of IPF, the ultimate treatment paradigm will likely comprise multiple therapies administered in combination," said Paul Noble, M.D., Professor of Medicine at Yale University. "Pirfenidone may hold the potential to be an important component of a treatment regimen aimed at this deadly disease."