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FDA Grants Orphan Drug Designation to Omeros’ MASP-3 Inhibitor OMS906 for Treatment of Paroxysmal Nocturnal Hemoglobinuria
-- Enrollment of PNH Patients Expected to Begin this Summer -- SEATTLE--(BUSINESS WIRE)-- Omeros Corporation today announced that OMS906 has received orphan
About this update from Omeros Corporation
[{"type":"text","content":"\n-- Enrollment of PNH Patients Expected to Begin this Summer --\n\n SEATTLE--(BUSINESS WIRE)--\nOmeros Corporation today announced that OMS906 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). OMS906 targets mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the alternative pathway of the complement system. MASP-3 converts pro-complement factor D (pro-CFD) to mature CFD.\n\nPNH is a rare, life-threatening disease characterized by red blood cell destruction, blood clots and impaired bone marrow function. Based on its mechanism of action as well as the pharmacokinetic/pharmacodynamic (PK/PD) profile shown in a completed Phase 1 study, OMS906 has the potential to offer a favorable safety profile and more convenient dosing than other drugs on the market or in development for PNH. Also, different than other enzymes in the alternative pathway targeted by competitors’ agents, MASP-3 does not appear to be an acute phase reactant, meaning that the concentration of MASP-3 – and the effective dosing level of OMS906 – do not change in the setting of inflammation. Omeros continues to build a strong and exclusive intellectual property position around therapeutics targeting MASP-3.\n\n“We are excited to begin clinical studies to demonstrate the efficacy of OMS906 in alternative pathway-related disorders,” said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. “Based on our preclinical and Phase 1 trial data, we expect that OMS906 will perform well. With the potential advantages of MASP-3 inhibition and OMS906 – decreased infection risk, better dosing profile, and the ability to avoid ‘breakthrough’ disease seen with agents targeting acute phase reactants – we believe that OMS906 could become first-line therapy in alternative pathway disorders.”\n\nFDA 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 dru...