Omeros Gets Orphan Status for PNH Therapy

FDA has granted Omeros an orphan drug designation for OMS906 and its use in treating paroxysmal nocturnal hemoglobinuria (PNH). The rare, life-threatening disease is characterized by red blood cell destruction, blood clots and impaired bone marrow function.

The company says that based on OMS906’s mechanism of action as well as the pharmacokinetic/pharmacodynamic (PK/PD) profile shown in a completed Phase 1 study, the therapy may offer a favorable safety profile and more convenient dosing than other drugs on the market or in development for PNH.

OMS906 is said to target mannan-binding lectin-associated serine protease-3 (MASP-3), a key activator of the alternative pathway of the complement system. “MASP-3 converts pro-complement factor D (pro-CFD) to mature CFD,” according to the company. “Based on the results of the completed Phase 1 trial, administration of OMS906 is expected to be once monthly to once quarterly intravenously or subcutaneously,” it adds.