Omeros Corporation announced that OMS721 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Immunoglobulin A (IgA) nephropathy. 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. IgA nephropathy is the most common primary glomerulopathy globally, with an estimated 120,000 to 180,000 cases in the U.S. alone, and accounts for up to 10 percent of all dialysis patients. Up to 40 percent of individuals with the disease develop end-stage renal disease, a life-threatening condition, within 20 years following diagnosis.
As previously reported, Phase 2 clinical trial results with OMS721 in IgA nephropathy patients show unprecedented reductions in urine protein levels during and following treatment with OMS721. Elevated urinary protein is highly correlated with poor outcomes in patients with IgA nephropathy. Following review of these data, FDA in June granted OMS721 breakthrough therapy designation for the treatment of IgA nephropathy. Omeros plans to begin enrolling patients in its Phase 3 registration trial in IgA nephropathy later this year.
“Working with FDA, we are initiating another Phase 3 program for OMS721 – this one in IgA nephropathy, which has been granted both breakthrough and orphan designations,” stated Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. “This marks the second Phase 3 program for OMS721, joining our ongoing aHUS program that already has received fast track status from FDA. A third Phase 3 program for OMS721 could be added as well this year in stem cell transplant-associated thrombotic microangiopathy that, together with aHUS, has been granted orphan designation. Focused on bringing OMS721 to market as quickly as possible, we are excited about its prospects and the benefits that we expect OMS721 will provide for patients across a wide range of serious and life-threatening disorders.”
FDA grants orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments that target a condition affecting 200,000 or fewer U.S. patients annually. It qualifies a company for benefits that apply across all stages of drug development, including seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and waiver of certain administrative fees.
To date, more than 150 subjects worldwide have received OMS721, and no safety concerns have been noted.