Omeros Corporation announced today that the European Medicines Agency's (EMA's) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending orphan drug designation of OMS721 for treatment in hematopoietic stem cell transplantation. 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. Three OMS721 Phase 3 clinical programs are progressing across hematopoietic stem cell transplant-associated thrombotic microangiopathy (TMA), immunoglobulin A (IgA) nephropathy, and atypical hemolytic uremic syndrome (aHUS).
The positive opinion is expected to be published by COMP within the week and to be adopted by the European Commission in August. 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 European Union (EU). This designation allows for financial and regulatory incentives that include 10 years of marketing exclusivity in the EU after product approval, reduced EMA advisory, inspection and filing fees pre- and post-approval, and guaranteed access to centralized marketing authorization valid in all EU member states as well as in the European Economic Area countries (i.e., Iceland, Liechtenstein and Norway).
The U.S. Food and Drug Administration (FDA) earlier granted orphan designation to OMS721 for the prevention (inhibition) of complement-mediated TMA and, in April of this year, granted breakthrough therapy designation to OMS721 for the treatment of stem cell-transplant patients who have persistent TMA despite modification of immunosuppressive therapy. Discussions are ongoing with both U.S. and European regulators regarding expedited pathways to approval for OMS721 in the treatment of "high-risk" stem cell transplant-associated TMA.