Discovery Laboratories, Inc. (Nasdaq:DSCO), Warrington, Penn., has announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has granted orphan drug designation to Discovery Labs' KL4 surfactant for the treatment of cystic fibrosis (CF). Orphan designation provides for up to ten years of market exclusivity in the European Union (EU) for the drug product with the designated orphan indication. This period of market exclusivity initiates upon EU marketing authorization.
Dr. Thomas F. Miller, Discovery Labs' Chief Operating Officer commented, "Discovery Labs has successfully procured orphan designations for several respiratory disease targets for our KL4 surfactant. We now have successfully secured orphan designations for CF treatment in both the U.S. and Europe. Our development experience to date suggests that CF may be a viable therapeutic target for our aerosolized KL4 surfactant technology."
CF is caused by a genetic mutation that leads to the production of thick, viscous mucus that is difficult to clear from the airways of the lung. The abnormal mucus allows for chronic airway infections that lead to airway destruction, decreased lung function, and ultimately, death. Last year, Discovery Labs announced the completion of an investigator-initiated, double-blind, randomized crossover Phase 2a study that evaluated the safety, tolerability and effectiveness of aerosolized KL4 surfactant in a CF patient population. The trial results were presented at the 2010 North American Cystic Fibrosis Conference, and the principal investigator concluded that aerosolized KL4 surfactant delivery to CF patients was feasible, was generally safe and well-tolerated, and was not associated with serious adverse events.
About Orphan Designation (US and EU)
Orphan designation by the EMA is intended to encourage sponsor development of new therapeutics for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions affecting no more than five in 10,000 people in the EU for which there is no satisfactory method of diagnosis, prevention or treatment of the condition at time of market authorization. Receipt of an orphan designation by EMA qualifies the sponsor for up to ten years of marketing exclusivity following marketing authorization, as well as other sponsor incentives including reduction in certain fees typically associated with the drug product review process in the EU. The U.S...