An orphan drug designation confers a number of benefits including ten years of potential market exclusivity if the product candidate is approved for marketing in the EU, and regulatory assistance in preparing the marketing application.
Cystic fibrosis (CF) is a fatal disease involving a genetic mutation that disrupts the cystic fibrosis transmembrane regulator (CFTR) protein. This protein acts as an ion-specific channel that moves salt and water to the surface of the airways.
Denufosol tetrasodium is designed to enhance the lung’s innate mucosal hydration and mucociliary clearance mechanisms by activating an alternative ion channel that acts in the same way as the defective ion channel in moving salt and water to the surface of the airways.
This unique approach is different from the approach of other approved CF products and may be important in intervening in the early clinical course of CF lung disease.
“We are pleased to make continued progress in our CF program and we look forward to using the assistance available through the EMEA’s Orphan Drug program,” said Dr Christy Shaffer, CEO of Inspire.
In March 2001, the FDA granted orphan drug status for denufosol for the treatment of CF in the US.