Omeros Corporation (NASDAQ: OMER) today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to OMS721 for the treatment of patients with atypical hemolytic uremic syndrome (aHUS). OMS721 is the company's lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the key regulator of the lectin pathway of the immune system. Omeros is currently evaluating the drug in a Phase 2 clinical trial for the treatment of aHUS and other thrombotic microangiopathies (TMAs). TMAs are a family of rare, debilitating and life-threatening disorders characterized by excessive thrombi (clots) in the microcirculation of the body's organs, most commonly the kidney and brain. Earlier this year, Omeros announced positive data from the Phase 2 trial and commencement of an investigator-requested compassionate use program for OMS721 to allow extended treatment of patients who completed the trial's four-week dosing. Recent efficacy and safety data from the ongoing Phase 2 clinical trial were submitted to the FDA in the company's request for Fast Track status for OMS721.
FDA's Fast Track program facilitates the development of drugs intended to treat serious or life-threatening conditions and that have the potential to address unmet medical needs. A drug program with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval. Many drugs that receive Fast Track designation are also considered appropriate to receive Priority Review, and their respective New Drug Applications (NDAs) may be accepted by the FDA as a "rolling submission" in which portions of an NDA are reviewed before the complete application is submitted. Priority Review and rolling submission can each provide further acceleration of FDA's approval process.
"FDA's Fast Track designation of OMS721 for aHUS reflects the unmet need associated with this disease and recognizes the drug's potential as an important option for the treatment of aHUS," stated Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "We look forward to working with the FDA to streamline the development of this promising drug, and we currently remain on track to discuss with the Agency later this year both the data from our Phase 2 trial in TMAs as well as our plans for our Phase 3 program."
The Phase 2 clinical trial evaluating OMS721 in the treatment of TMAs, including aHUS, thrombotic thrombocytopenic purpura and human stem cell transplant-related TMAs, is ongoing in multiple sites in both the United States and Europe. The majority of the patients enrolled to date are those suffering from aHUS.