7 August 2017 - - The US Food and Drug Administration has granted Seattle, Washington-based small-molecule and protein therapeutics specialist Omeros Corp. (NASDAQ: OMER) orphan drug designation for OMS721 for the treatment of Immunoglobulin A (IgA) nephropathy, the company said.
OMS721 is Omeros' lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.
Phase 2 clinical trial results with OMS721 in IgA nephropathy patients show unprecedented reductions in urine protein levels during and following treatment with OMS721. Following review of these data, FDA in June granted OMS721 breakthrough therapy designation for the treatment of IgA nephropathy.
Omeros plans to begin enrolling patients in its Phase 3 registration trial in IgA nephropathy later this year.
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 US patients annually.
To date, more than 150 subjects worldwide have received OMS721, and no safety concerns have been noted.
Following discussions with both the FDA and the European Medicines Agency, a Phase 3 clinical program for OMS721 in atypical hemolytic uremic syndrome is in progress.
The company has established a compassionate-use program for OMS721, which is active in both the US and Europe.
Omeros is committed to discovering, developing and commercialising both small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, coagulopathies and disorders of the central nervous system.
It has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-associated thrombotic microangiopathies, complement-mediated glomerulonephropathies, Huntington's disease and cognitive impairment, and addictive and compulsive disorders.
The company has a proprietary G protein-coupled receptor platform and controls 54 new GPCR drug targets and corresponding compounds, a number of which are in preclinical development. It also exclusively possesses a novel antibody-generating platform.