– Preparations Continue for Submission of BLA in the U.S. and MAA in Europe –
Omeros Corporation (OMER) today announced that the European Medicines Agency (EMA) has appointed rapporteurs for the company’s marketing authorization application (MAA) currently in preparation for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Narsoplimab, also known as “OMS721,” is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) and is in Phase 3 clinical programs for the treatment of HSCT-TMA, immunoglobulin A nephropathy, and atypical hemolytic uremic syndrome.
The appointed rapporteurs are members of EMA’s Committee for Human Medicinal Products (CHMP) and will jointly coordinate CHMP’s evaluation of Omeros’ planned MAA for narsoplimab. Omeros intends to request a pre-submission meeting with the rapporteurs to discuss the planned submission of the MAA. As previously announced, narsoplimab for HSCT-TMA is eligible for submission under EMA’s centralized review procedure, which allows submission of a single MAA that, if approved, authorizes the product to be marketed in all 31 member states comprising the European Economic Area rather than requiring separate national approvals. Narsoplimab also has orphan drug designation from EMA for treatment in hematopoietic stem cell transplantation. Omeros is preparing both a U.S. biologics license application (BLA) and a European MAA for narsoplimab for the treatment of HSCT-TMA.
About Omeros’ MASP Programs
Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2, a novel pro-inflammatory protein target involved in activation of the complement system, which is an important component of the immune system. The complement system plays a role in the inflammatory response and becomes activated as a result of tissue damage or microbial infection. MASP-2 is the effector enzyme of the lectin pathway, one of the principal complement activation pathways. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection, and its abnormal function is associated with a wide range of autoimmune disorders. MASP-2 is generated by the liver and is then released into circulation. Gene-targeted MASP-2-deficient mice and humans with MASP-2 gene polymorphisms that affect MASP-2 serum levels and MASP-2 functional activity are generally healthy with no obvious adverse phenotype.
Phase 3 clinical programs are in progress for narsoplimab, Omeros’ lead MASP-2 inhibitor also known as “OMS721,” in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). Narsoplimab can be administered both intravenously and subcutaneously, and Omeros expects to commercialize each formulation of narsoplimab for different therapeutic indications. In parallel, Omeros is developing small-molecule inhibitors of MASP-2. Based on requests from treating physicians, Omeros has established a compassionate-use program for narsoplimab, which is active in both the U.S. and Europe. The FDA has granted narsoplimab breakthrough therapy designation for HSCT-TMA and IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.
Omeros also has identified MASP-3 as the key activator of the human complement system’s alternative pathway and as the enzyme responsible for the conversion of pro-factor D to factor D. The alternative pathway is linked to a wide range of immune-related disorders. In addition to its lectin pathway inhibitors, the company is advancing its development of antibodies and small-molecule inhibitors against MASP-3 to block activation of the alternative pathway. Omeros is scaling up the manufacturing of its MASP-3 antibodies in preparation for clinical trials slated for next year. In addition to its MASP-2- and MASP-3-specific programs, Omeros is developing bi-specific inhibitors of MASP-2/MASP-3.
About Omeros Corporation
Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. The company’s drug product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1% / 0.3% is marketed for use during cataract surgery or intraocular lens (IOL) replacement to maintain pupil size by preventing intraoperative miosis (pupil constriction) and to reduce postoperative ocular pain. In the European Union, the European Commission has approved OMIDRIA for use in cataract surgery and other IOL replacement procedures to maintain mydriasis (pupil dilation), prevent miosis (pupil constriction), and to reduce postoperative eye pain. Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on: complement-associated thrombotic microangiopathies; complement-mediated glomerulonephropathies; cognitive impairment; and addictive and compulsive disorders. In addition, Omeros has a diverse group of preclinical programs and a proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and corresponding compounds, a number of which are in preclinical development. The company also exclusively possesses a novel antibody-generating platform.
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