The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Omeros Corporation’s OMER lead asset OMS906 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
OMS906 is evaluated in the Phase 1 clinical in healthy subjects. The company expects to begin enrolment of PNH patients this summer.
Gregory A. Demopulos, Chairman and Chief executive officer, commented: "We are excited to begin clinical studies to demonstrate the efficacy of OMS906 in alternative pathway-related disorders. 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.”
The company is anticipating efficacy data readout in populations of treatment-naïve PNH and C3 glomerulopathy patients by early 2023.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease designated by red blood cell destruction, blood clots and impaired bone marrow function.
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.
Price Action : Omeros shares are trading around 15 percent higher at $5.12 on Friday at the time of publication.
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