Orphan Status for Omeros Immunoglobulin A Nephropathy Drug

FDA has granted Omeros an orphan drug designation for OMS721 for treating Immunoglobulin A (IgA) nephropathy. The human monoclonal antibody is designed to target mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system, according to the company. 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% of all dialysis patients, it says. Up to 40% of individuals with the disease develop end-stage renal disease.

The orphan designation is based on Phase 2 clinical trial data results showing significant reductions in urine protein levels during and following treatment with the therapy. Elevated urinary protein is highly correlated with poor outcomes in patients with IgA nephropathy, Omeros says.