Omeros Files Orphan Drug Application with FDA for OMS721

Omeros Corporation

today announced that it has filed an Application for Orphan Drug Designation with the U.S. Food and Drug Administration (FDA) for OMS721, the lead human monoclonal antibody in Omeros' mannan-binding lectin-associated serine protease-2 (MASP‑2) program, for use in the treatment of atypical hemolytic uremic syndrome (aHUS). As Omeros previously announced, based on positive preclinical data in thrombotic microangiopathy (TMA), the first indication planned for OMS721 clinical trials is aHUS, a rare but life-threatening form of TMA. Orphan drug designation is granted to treatments that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients per year. Orphan-designated drugs are eligible for incentives such as a faster approval process and additional market exclusivity. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2, a novel pro-inflammatory protein involved in activation of the complement system – an important component of the immune system. The complement system plays a role in the inflammatory response to tissue damage or microbial infection. OMS721 selectively inhibits MASP-2, blocking the lectin pathway of