Omeros Corporation initiates BLA rolling submission for narsoplimab for the treatment of HSCT-TMA.

Omeros Corporation has submitted the first sections of the rolling submission of its Biologics License Application (BLA) to the FDA for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a far-too-often deadly complication of stem-cell transplantation.

Narsoplimab, also known as OMS 721, is Omeros� lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2). There currently is no approved product for the treatment or prevention of HSCT-TMA, and narsoplimab is the only drug in development for the treatment of HSCT-TMA with FDA�s breakthrough therapy designation. Narsoplimab also has been granted FDA�s orphan drug designation for this indication.

Last month, Omeros announced that FDA had agreed with the company�s proposed schedule for the rolling review of its BLA for narsoplimab in the treatment of HSCT-TMA. Rolling submission enables Omeros to submit sections of the BLA as they are completed, which can accelerate the time to approval by allowing FDA to review the sections as they are submitted rather than waiting to begin its review until the entire BLA has been submitted.

Comment: There are approximately 25,000 to 30,000 allogeneic stem-cell transplantations in the U.S. and Europe annually. TMA has been reported to occur in approximately 40 percent of them, with an estimated 80 percent of those having high-risk features. The mortality rate in severe HSCT-TMA can exceed 90 percent.