RA Pharmaceuticals receives orphan drug designation from FDA for RA101495
Ra Pharmaceuticals announced that the U.S. FDA has granted Orphan Drug Designation to RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria. Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases. RA101495, the company's lead clinical candidate, is a synthetic macrocyclic peptide inhibitor of complement component 5. The molecule is currently in Phase 2 clinical development as a self-administered subcutaneous injection for the treatment of PNH, a rare, chronic, life-threatening blood disorder where red blood cells are attacked and destroyed by the complement system. RA101495 received Orphan Drug Designation for the treatment of PNH in Europe in November 2016.