Research & Development
FDA Grants Orphan Drug Designation to Omeros' MASP-3 Inhibitor OMS906 for Treatment of Paroxysmal Nocturnal Hemoglobinuria
29 July 2022 - - US-based biopharmaceutical company Omeros Corp's OMS906 has received orphan drug designation from the US Food and Drug Administration for the treatment of paroxysmal nocturnal hemoglobinuria, the company said.

OMS906 targets mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the alternative pathway of the complement system.

MASP-3 converts pro-complement factor D (pro-CFD) to mature CFD.

PNH is a rare, life-threatening disease characterized by red blood cell destruction, blood clots and impaired bone marrow function.

Based on its mechanism of action as well as the pharmacokinetic/pharmacodynamic (PK/PD) profile shown in a completed Phase 1 study, OMS906 has the potential to offer a favorable safety profile and more convenient dosing than other drugs on the market or in development for PNH.

Also, different than other enzymes in the alternative pathway targeted by competitors' agents, MASP-3 does not appear to be an acute phase reactant, meaning that the concentration of MASP-3 and the effective dosing level of OMS906 do not change in the setting of inflammation.

Omeros continues to build a strong and exclusive intellectual property position around therapeutics targeting MASP-3.

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.

It qualifies a company for benefits that apply across all stages of drug development, including seven years of market exclusivity following marketing approval, tax credits on US clinical trials, eligibility for orphan drug grants, and waiver of certain administrative fees.

Omeros recently completed a Phase 1 trial of OMS906 in healthy subjects and expects to begin enrollment this summer in a clinical trial assessing OMS906 in PNH patients who have had an unsatisfactory response to the C5 inhibitor ravulizumab.

The company is also targeting efficacy data in populations of treatment-naïve PNH and C3 glomerulopathy patients by early 2023.

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.

OMS906 is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement system's alternative pathway.

The complement system plays a central role in inflammation and becomes activated as a result of tissue damage or microbial infection.
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