Omeros Corporation have announced that their novel pro-inflammatory protein target, OMS721, has received Orphan Drug designation for the treatment of Immunoglobulin A (lgA) nephropathy from the Food and Drug Administration (FDA). 

OMS721 specifically targets mannan-binding lectin-associated serine protease-2 (MASP-2). The Orphan Drug announcement quickly follows the Breakthrough Therapy designation OMS721 received in June. 

Phase 2 trials have shown to be associated with reductions in urine protein levels following treatment with OMS721 in patients with lgA nephropathy.

The company is planning to start enrolling for a Phase 3 trial later this year. “This marks the second Phase 3 program for OMS721, joining our ongoing aHUS (atypical hemolytic uremic syndrome) program that already has received fast track status from the FDA,” said Gregory A. Demopulos, MD, CEO of Omeros.  

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MASP-2 is the effector enzyme of the lectin pathway of the complement system. Omeros controls the rights to MASP-2 and the therapies that target it. As well as being granted Orphan Drug status for the indications of lgA nephropathy and aHUS, OMS721 has also been granted Orphan status for in stem cell transplant-associated thrombotic microangiopathy.

“We are excited about its (OMS721) prospects and the benefits that we expect OMS721 will provide for patients across a wide range of serious and life-threatening disorders,” said Dr. Demopulos. 

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