Omeros Receives Orphan Designation in Europe for Treatment in Hematopoietic Stem Cell Transplantation

CP Staff
CP Staff
  • OMS721 is also being studied in atypical hemolytic uremic syndrome, IgA nephropathy, and lupus nephritis
  • The drug also has orphan status for the treatment of primary IgA nephropathy


Omeros Corporation (Nasdaq: OMER) announced on 8/28/18 that the European Commission (EC) has adopted a decision designating OMS721 as an Orphan Medicinal Product in the European Union (EU) for treatment in hematopoietic stem cell transplantation (HSCT) associated thrombotic microangiopathy. The adoption by the EC follows a positive opinion for orphan designation of OMS721 in this indication by the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system. There are three Phase 3 development programs ongoing with OMS721 in each of HSCT-associated thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS), and immunoglobulin A (IgA) nephropathy. Thrombotic microangiopathy (TMA) is a pathology that results in thrombosis in capillaries and arterioles, due to an endothelial injury.

This represents the second orphan drug designation in Europe for OMS721, with the first being for OMS721 in the treatment of primary IgA nephropathy. Orphan drug designation in Europe is available to companies developing products intended to treat a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 persons in the EU. This designation allows for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase, and access to centralized marketing authorization. OMS721 also has been granted orphan designations from the U.S. Food and Drug Administration (FDA) for indications related to both HSCT-associated TMA and IgA nephropathy.

Omeros is in discussions with European regulators and FDA to discuss full and conditional/accelerated approval for OMS721 in HSCT-associated TMA.