Discovery Laboratories, Inc. announced that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted orphan drug designation to Discovery's lead product, Surfaxin, for the prevention of Bronchopulmonary Dysplasia (BPD, also known as Chronic Lung Disease) in premature infants.
Surfaxin, a precision-engineered lung surfactant replacement therapy, has previously received orphan drug designation for the treatment of BPD, as well as fast track designation for both the prevention and treatment of BPD.
BPD is a costly syndrome affecting premature infants. It is associated with surfactant deficiency and the prolonged use of mechanical ventilation and oxygen supplementation. Some premature babies are born with a lack of natural surfactant in their lungs. Without surfactant, the air sacs in the lungs collapse and are unable to absorb sufficient oxygen resulting in Respiratory Distress Syndrome (RDS). To prevent and treat RDS, babies require a surfactant usually within the first hours of birth, and mechanical ventilation to support their respiration. The lack of surfactant and use of mechanical ventilation may cause chronic injury and scarring of the lungs -- resulting in BPD.
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. Orphan drug designation in the United States is awarded to compounds that offer potential therapeutic value in the treatment of rare diseases, defined as those affecting fewer than 200,000 Americans. If the company complies with certain FDA specifications and should the drug receive marketing approval, orphan drug designation qualifies the sponsor for seven years of marketing exclusivity, exemption from the Prescription Drug User Fee Act filing fees, and tax credits related to clinical research.
Robert J. Capetola, Ph.D., president and chief executive officer of Discovery commented, "Market exclusivity under this designation would mean that Surfaxin, as a precision-engineered surfactant, has the potential to become the dominant surfactant in the neonatal intensive care unit for the next decade. If Surfaxin is the first product to receive marketing authorization in the United States for the treatment of Bronchopulmonary Dysplasia, orphan status would block any similar synthetic surfactant products for this indication throughout the United States market for a significant period of time."
Discovery recently concluded its phase 2 double-blind, placebo-controlled trial early, enrolling approximately 130 very low birth weight premature infants born at risk for developing BPD. The purpose of the trial was to determine the safety and tolerability of administering Surfaxin as a therapeutic approach for the prevention and treatment of BPD. Premature infants in this study received a treatment regime of up to 5 Surfaxin doses beginning within the first 3-10 days of life, in addition to the surfactant they received on day 1 of life for RDS. Discovery plans to report the top-line results in the fourth quarter of 2006.
Surfaxin is a precision-engineered, peptide-containing, synthetic surfactant that is designed to closely mimic the function of natural human lung surfactant. Discovery's Surfaxin has received two Approvable Letters from the FDA for the prevention of RDS in premature infants. Surfactants are substances that are produced naturally in the lungs and are essential to the lungs' ability to absorb oxygen and to maintain proper airflow through the respiratory system. Premature babies are born with a lack of natural surfactant in their lungs. Without surfactant, the air sacs in the lungs collapse and are unable to absorb sufficient oxygen resulting in RDS.