Omeros submits pediatric investigational plan for use of OMS721 to EMA

Omeros Corporation announced that it has submitted a pediatric investigational plan for the use of OMS721 for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy to the European Medicines Association. A pediatric study plan is also under development for submission to the U.S. Food and Drug Administration. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2, the effector enzyme of the lectin pathway of the complement system. OMS721 was awarded breakthrough therapy designation for the treatment of high-risk HSCT-TMA earlier this year. Thrombotic microangiopathy is a life-threatening complication of HSCT, with mortality reported to be greater than 90 percent in high-risk patients. In addition to the data in adult HSCT-TMA patients forming the basis for its U.S. Biologics License Application (BLA) and E.U. Marketing Authorization Application currently in preparation, Omeros is proposing a plan including a small study in pediatric patients that will accelerate development of OMS721 for the treatment of HSCT-TMA in children. Rather than deferring initiation of a pediatric clinical trial until after approval in the E.U. or foregoing the study in the U.S., which is an option for an orphan drug, Omeros is proposing to initiate assessment of OMS721 in the pediatric population prior to approval in view of the strong OMS721 data observed to date and the significant unmet medical need. Because OMS721 has been designated as an orphan medicinal product in the EU, successful completion of an agreed PIP will provide two additional years of market exclusivity in member states of the European Union, and in Norway, Liechtenstein, and Iceland. Omeros is also developing a PSP for submission to the FDA. Successful completion of an agreed PSP in response to a Written Request from FDA provides an additional 6 months of market exclusivity in the United States. OMS721 also has been granted orphan drug designation for the treatment of HSCT-TMA by the FDA. Although the requirement for pediatric studies is waived for drugs with orphan drug designation, pursuing a PSP can help accelerate pediatric treatment in the U.S. and provide additional market exclusivity. A large prospective study reported that approximately 40% of pediatric patients who undergo HSCT will develop TMA and approximately 80% of these patients will have high-risk features. Omeros has also received notification from EMA of eligibility for the centralized procedure for submission and review of its MAA for OMS721 in the treatment of HSCT-TMA. The EMA’s centralized procedure allows submission of a single MAA that, when approved, authorizes the drug to be marketed in all European Union member states and European Free Trade Association countries rather than requiring separate national approvals.
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