OMS721 Designated Orphan Drug for Thrombotic Microangiopathies

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.

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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.

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