Orphan drug designation has been granted to OMS721, a product of Omeros Corporation, from the U.S. Food and Drug Administration (FDA) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). OMS721 is human monoclonal antibody that targets the enzyme - mannan-binding lectin-associated serine protease-2 (MASP-2). This enzyme has significant governing role in the working of the lectin pathway of the complement system. Phase 3 clinical programs are in progress for OMS721 in atypical hemolytic uremic syndrome (aHUS), in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) and in immunoglobulin A nephropathy.
Earlier in 2018, OMS721 had been granted breakthrough therapy designation for the treatment of high-risk HSCT-TMA. Thrombotic microangiopathy is a fatal complication of HSCT, and some high-risk patients can face a mortality risk of more than 90%. Gregory A. Demopulos, M.D., chief executive officer and chairman of Omeros expressed contentment over the agency's move. With orphan designations that cover both the prevention and treatment of HSCT-TMA with OMS721 in the U.S. as well as in Europe, he reiterated the company's vision of saving maximum lives by making the drug available to transplanters and their patients in the within the shortest window of time.
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