Omeros Corporation has filed an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for OMS721 in the treatment of immunoglobulin A (IgA) nephropathy (also known as Berger’s disease). IgA nephropathy leads to end-stage renal disease in up to 40 percent of patients. Orphan drug designation is granted to treatments that target conditions affecting 200,000 or fewer U.S. patients per year. Orphan-designated drugs are eligible for financial incentives and regulatory advantages such as a faster approval process and additional market exclusivity. 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.
FDA earlier granted orphan designation for OMS721 in thrombotic microangiopathies (TMAs), including atypical hemolytic uremic syndrome (aHUS) and hematopoietic stem cell-associated TMA (HSCT-TMA). Based on positive Phase 2 data in renal diseases, Omeros also recently filed for FDA fast track designation for OMS721 in IgA nephropathy. The company already has received fast track designation for OMS721 in aHUS.
In addition to positive Phase 2 data in IgA and membranous nephropathies, Omeros has reported positive data from Phase 2 clinical trials in both aHUS and HSCT-TMA. Following guidance from FDA and from the European Medicines Agency, Omeros plans to open enrollment for its single Phase 3 trial in patients with aHUS later this year. Based on discussions with the FDA, the company is pursuing breakthrough therapy designation for OMS721 in IgA nephropathy and in HSCT-TMA, neither of which has any approved treatment. Omeros plans to initiate OMS721 Phase 3 programs in both IgA nephropathy and HSCT-TMA next year.