The Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted orphan drug designation to Discovery Laboratories' lead product, Surfaxin, for the treatment of bronchopulmonary dysplasia (also known as Chronic Lung Disease, CLD) in premature infants. Surfaxin, a precision-engineered lung surfactant replacement therapy, has received an approvable letter from the US FDA for the prevention of Respiratory Distress Syndrome (RDS) in premature infants and is pending approval.
CLD is a costly syndrome associated with surfactant and SP-B deficiency, and the prolonged use of mechanical ventilation and oxygen supplementation, usually associated with a premature infant being treated for RDS. Presently there are no approved drugs for the treatment of CLD. These babies suffer from abnormal lung development and typically have a need for respiratory assistance at times, for many months, as well as comprehensive care that sometimes can span many years, claims a company release.
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 (PDUFA) filing fees, and tax credits related to clinical research.
Robert J. Capetola, 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 engineered surfactant 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 future similar products for this indication throughout the United States market for a prolonged period of time."
Discovery is currently conducting a Phase 2 double-blind, controlled trial (that will enroll up to 210 very low birth weight premature infants born at risk for developing CLD) to determine the safety and tolerability of administering up to 5 total doses of Surfaxin in the first 10 days of life as a therapeutic approach for the prevention and treatment of CLD. This study is designed to determine whether such treatment can decrease the proportion of infants on mechanical ventilation or oxygen or the incidence of death or CLD.
Surfaxin is a precision-engineered, peptide-containing, synthetic surfactant that is designed to closely mimic the function of natural human lung surfactant.