Discovery Laboratories Inc announced that the Commission of the European Communities has designated Surfaxin as an Orphan Medicinal Product.
According to a release, this designation is for the prevention of Respiratory Distress Syndrome (RDS) in premature neonates of less than 32 weeks of gestational age and for the treatment of RDS in premature neonates of less than 37 weeks of gestational age. Designation was granted following the positive opinion given by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Evaluation Agency (EMEA) in which the COMP concluded that although satisfactory methods of prevention and treatment of RDS have been authorized in Europe, justifications have been provided that Surfaxin may be of significant benefit to those at risk of developing or affected by the condition.
This designation allows Discovery exclusive marketing rights for Surfaxin in the indication of RDS in Europe for ten years (subject to revision after six years) following marketing approval by the EMEA. In addition, the designation enables Discovery to receive regulatory assistance in the further development process of the drug, and to access reduced regulatory fees throughout its marketing life.
Christopher J. Schaber, executive vice president and chief operating officer of Discovery, stated, "The granting of Orphan Medicinal Product designation together with the United States FDA's acceptance of the Surfaxin NDA, increases our confidence in the potential for Surfaxin to become a new, worldwide standard of care for the prevention and treatment of RDS. Market exclusivity under this designation would equate to Surfaxin becoming the only engineered surfactant available in Europe. With this designation and our existing patent portfolio, we believe Surfaxin has the potential to become the dominant engineered surfactant for the next decade and thereby block any future similar products for the treatment and prevention of RDS throughout the European market."
The US FDA has already granted orphan drug designation for Surfaxin for the prevention of RDS in premature infants. Recently the FDA accepted Discovery's New Drug Application for Surfaxin for the prevention of RDS in premature infants and has granted a PDUFA date of February 13, 2005. Discovery also is preparing a Marketing Authorization Application (MAA) to be filed with the EMEA for Surfaxin for the prevention and treatment of RDS.
Discovery's Surfaxin is an engineered version of natural human lung surfactant and contains a peptide, sinapultide, that is designed to closely mimic the essential human lung surfactant protein B (SP-B). Surfaxin, unlike the animal products, can be produced in virtually unlimited quantities, in consistent pharmaceutical grade quality, and has no risk of potential transmission of animal-associated diseases.
The EMEA grants orphan drug designation to medicinal products based upon several criteria: the life-threatening and debilitating nature of the condition; the medical plausibility of the proposed orphan indication; a prevalence in Europe of less than 5 cases for each 10,000 of population; and the lack of a satisfactory method of diagnosis, prevention or treatment or, if such method exists, the medicinal product will be of significant benefit to those affected by that condition.