Omeros announced that the FDA has granted Orphan Drug designation to OMS721 for the prevention of complement-mediated thrombotic microangiopathies (TMAs). OMS721 is a human monoclonal antibody that selectively inhibits mannan-binding lectin-associated serine protease-2 (MASP-2), thus blocking the lectin pathway of the complement system while leaving the classical pathway intact.
RELATED: Hematological Disorders Resource Center
Omeros is currently completing a Phase 1 study to assess the safety and pharmacokinetics of OMS721. At the highest subcutaneous dose administered to date in this study, OMS721 achieved serum concentrations that resulted in a high degree of inhibition of lectin pathway activation. The Phase 2 clinical program evaluating OMS721 for the prevention of complement-mediated TMAs is expected to begin in the first quarter of 2014.
TMAs, including atypical hemolytic uremic syndrome and thrombotic thrombocytopenic purpura, are a family of rare, debilitating and life-threatening disorders characterized by multiple thrombi in the microcirculation of the body’s organs, most commonly the kidney and brain.
For more information call (206) 676-5000 or visit Omeros.com.