FDA Grants Breakthrough Therapy Designation to OMS721 for IgA Nephropathy

The FDA has granted breakthrough therapy designation to OMS721, a new treatment for the rare kidney disease immunoglobulin A (IgA) nephropathy. This designation is a significant milestone for Seattle-based Omeros, the developer of the drug, leading to a 14% surge in its share price.

OMS721 is Omeros’ lead human monoclonal antibody targeting MASP-2, a novel pro-inflammatory protein involved in the activation of the complement system, a crucial part of the immune system. It is the only candidate to receive FDA breakthrough designation for IgA nephropathy, based on phase 2 trial data showing unprecedented improvement in proteinuria, a key marker for disease progression.

Clinical trial data presented at the 54th European Renal Association-European Dialysis and Transplant Association Congress in Madrid demonstrated a 77% mean reduction in urine albumin-to-creatinine ratios and a 73% mean reduction in 24-hour urine protein levels after 12 weeks of OMS721 treatment. These results have sparked interest among physicians from Europe, the US, and Asia, who have expressed a desire to participate in the planned phase 3 trial.

Gregory A. Demopulos, M.D., chairman and CEO of Omeros, highlighted the potential of OMS721 to help IgA nephropathy patients with a rapidity and magnitude not seen with any other therapy. There is currently no approved treatment for IgA nephropathy, a condition that affects an estimated 120,000 to 180,000 patients in the US alone and accounts for up to 10% of all dialysis patients.

OMS721 is also being evaluated in phase 3 trials for atypical haemolytic uremic syndrome (aHUS) and other indications, including haematopoietic stem cell transplant-associated thrombotic microangiopathy, with a further phase 3 clinical programme in stem cell transplant-associated TMA planned to begin this year.