Discovery Laboratories Inc announced that the Committee for Orphan Medicinal Products (COMP) of the European Agency for the Evaluation of Medicinal Products (EMEA) has adopted a positive opinion recommending the granting of orphan medicinal product designation for Surfaxin for the prevention and treatment of Respiratory Distress Syndrome (RDS) in premature infants. In assessing Surfaxin for orphan drug designation, 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.
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.
Christopher J. Schaber, executive vice president and chief operating officer of Discovery, stated, "This positive opinion from the EMEA/COMP, together with the US 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."
If Surfaxin is granted orphan medicinal product designation by the COMP, Discovery would receive up to ten years of European market exclusivity effective upon approval of the Marketing Authorization Application (MAA) and also may be granted accelerated evaluation status in instances where there is an insufficiency of alternative therapeutics treating a disease or where the EMEA anticipates a high therapeutic benefit.
"Market exclusivity under this designation would equate to Surfaxin becoming the only engineered surfactant available in Europe. With this anticipated 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," commented Robert J Capetola, president and CEO of Discovery.
The COMP is a scientific body made up of representatives from the 25 member states of the European Union, Iceland, Norway and representatives of patient organizations. The Committee reviews orphan medicinal product designation applications on their scientific and clinical merit, and provides advice on their approval to the European Commission. A positive opinion is a critical step in obtaining an orphan medicinal drug designation from the European Commission for a product.
The US FDA has already granted orphan drug designation for Surfaxin for the prevention of RDS in premature infants in the United States. 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 MAA to be filed with the EMEA for Surfaxin for the prevention and treatment of RDS.
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.
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.
Discovery has filed a New Drug Application with the FDA for clearance to market Surfaxin, the Company's lead product, for the prevention of Respiratory Distress Syndrome in premature infants. Discovery is currently conducting a Phase 2 clinical trial for Acute Respiratory Distress Syndrome in adults, Phase 3 and Phase 2 clinical trials for Meconium Aspiration Syndrome in full-term infants and with aerosolized surfactant formulations is preparing to initiate a Phase 2 trial for asthma (development name DSC-104) and a Phase 2 trial for Respiratory Dysfunction in premature infants.