Ra Pharmaceuticals (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for diseases of complement dysregulation, today announced that the European Commission has designated RA101495 as an orphan medicinal product for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). RA101495, Ra Pharma's lead clinical candidate, is a synthetic macrocyclic peptide inhibitor of complement component C5. The molecule is currently in development as a self-administered subcutaneous injection for the treatment of PNH, a serious hematologic disease characterized by pathological red blood cell lysis due to unregulated activity of the complement system. "PNH is a rare and life-threatening blood disease for which new treatment options are desperately needed. We are pleased that the European Commission recognizes this urgency in granting orphan drug designation to RA101495," said Doug Treco, PhD, Co-Founder, President and CEO of Ra Pharma. "We are very encouraged by our Phase I data, which demonstrate that RA101495 is a potent inhibitor of C5-mediated hemolysis with a favorable safety and tolerability profile. We are developing RA101495 for convenient self-administration via subcutaneous injection, and we look forward to advancing through clinical development to make this innovative product available to patients with PNH." Orphan drug designation by the European Commission provides certain regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union, and for which no satisfactory treatment is available. In situations where there is already an approved standard of care, such as with PNH where the monoclonal antibody eculizumab (Soliris) is currently available, the European Commission requires sponsors to provide evidence that the product under consideration is expected to provide significant benefits over the standard of care. In the case of RA101495, the decision to grant orphan drug designation was based on the potential for improved patient convenience with subcutaneous self-administration, and the potential to treat patients that do not respond to eculizumab.